MORE PRIVATE FUNDS NEEDED IN BIOTECH

Posted May 5, 2010 by Sanjoy Gupta
Categories: Science

SEEING biotech as the sunrise industry, the Finance Minister, has granted several sops for R&D in the sector. But analysts and experts in the field believe the expected inflow of private funds into the sector could hardly meet the demand.

A recent study by Rabo India and the Confederation of Indian Industry (CII) on financing biotech enterprises says that the total private sector investment in Indian biotechnology industry till 2002 is estimated to be around $50 million (Rs 250 crore). “The requirement at the moment is however, far more, about $200-300 million,” said Mr Alok Gupta, Associate Director and Head- Life Sciences & Biotechnology at Rabo India Finance Pvt Ltd.

The Union Budget has granted biotechnology industry status at par with information technology and also duty waivers for research and development and clinical equipment.

In the last three years, biotechnology has been emerging as one of the most talked about sectors and the expectations are high. But the funds do not seem to be coming in. “After September 2001, many emerging businesses are having a difficult time raising money. This is because first-time entrepreneurs usually lack significant personnel; resources, bank loans are usually unavailable to businesses with no financial history, and venture capitalists are finding it difficult to raise enough money in a depressed market to invest in high-risk biotechnology ventures,” the report states.

Some of the private equity firms and venture capitalists that are active in the sector are ICICI Ventures, IL&FS, Westbridge Capital, UTI Venture, Canbank Venture Capital, IFCI Venture and Chrysalis Capital. Government funding both at the state and central levels have been found wanting. Although several schemes such as R&D and biotech funds have been announced, proper disbursal of the money is yet to take place.

The future seems to be rather bleak. Small biotech ventures are expected to die a natural death in the absence of private equity funding especially in the crucial second stage. “Entrepreneurs usually start up with what they have and with `angel investors’ chipping in. But once it has been accomplished, the second round of funding becomes crucial to carry on the work. Financial institutions are not comfortable in investing at this stage as there is no clarity on product pipeline,” said an industry source.

Also, competition will emerge from pure pharma research and development companies which will try and wrest some of the pie. Says Dr V.V.L.N. Sastry, Country-head, Firstcall Equity Advisors, “Investments have traditionally gone into the manufacturing firms and are now getting into biotechnology. But with the various sops in the latest Budget for pure R&D, biotechnology companies could be affected.”

The foray of big pharma companies such as Wockhardt and Ranbaxy into biotechnology has opened up another avenue.

“Pharma companies with biotech projects have a much better chance of attracting investments purely on the back of their proven performance. Areas that Indian companies have an upper-hand is contract research, manufacturing and clinical trials in the biotechnology industry,” said Mr Gupta.

There are Indian companies who are also looking for acquisitions of small biotech venture abroad to sustain its product pipeline.

Yogesh Agrawal & Sanjoy Gupta

Editor in Chief

Biotechtrove’s Blog

ICMR Recommends RS-GIS to control Vector Borne Disease

Posted May 4, 2010 by Sanjoy Gupta
Categories: Science

Remote sensing and geographical information system are expected to be an effective preventive measure against the emerging and re-emerging vector-borne diseases that pose great challenge to the researchers, disease control program planners and implementors.
The recent studies by the Indian Council of Medical Research (ICMR) task force to study the applications of the Remote Sensing (RS) and Geographical Information System (GIS) on the vector-borne disease prevention, has led to the conclusion that these technologies can be very effective in controlling the vector-borne diseases prevalent in our country. The major vector- borne diseases in India include malaria, filariasis, dengue, Japanese encephalitis (JE) and Visceral leishmaniasis, claiming millions of lives each year.
Recognizing the high potential of GIS and RS technologies in vector-borne diseases, the advances in RS with the availability of IRS-1C and 1D satellites that have a better resolution, and the developments in GIS technology, the Epidemiology and Communication Diseases Division of the ICMR for the first time initiated task force projects in vector-borne diseases using GIS and RS application tools. The projects were initiated in malaria, filariasis and visceral leishmaniasis.
The six member task force headed by Dr S Pattanayak recommended the use of GIS and RS are tools that can be used to provide solutions in vector-borne disease control programs. The studies highlighted the application of RS and GIS for the management of vector-borne disease by predicting the high-risk areas and the vulnerable periods of disease outbreaks.
Dr VM Katoch, Director General, ICMR, said, “The information will stimulate the professionals and academia to take up more studies in this area. The RS and GIS technology applications can be used to develop public healthcare strategies for all emerging and reemerging vector-borne diseases.”
GIS is an information technology tool comprising computer hardware and software to input, store, update, retrieve, analyze and output geo-referenced data. It has the potential to develop thematic maps of the input data and overlaying and integration of maps, and has a strong statistical component to visualize, interpret complex real world situations to provide effective solutions.

Yogesh Agrawal  &  Sanjoy Gupta
Editor in Chief
Biotech Trove’s Blog

Courtesy  :-  http://www.biospectrumasia.com/

THE ROLE OF PROTEIN IN PACKAGING DNA

Posted April 30, 2010 by Sanjoy Gupta
Categories: Science

Chromosome – The Role Of Proteins In Packaging Dna

Several kinds of proteins are important for maintaining chromosomes in terms of its organization and gene expression. Some proteins initiate DNA replication when the cell prepares to divide. Other proteins control gene transcription in the preliminary stages of protein synthesis. Structural proteins help the DNA fold into the intricate configurations within the packaged chromosome.

DNA in chromosomes is associated with proteins and this complex is called chromatin. Euchromatin refers to parts of the chromosome that have coding regions or genes, while heterchromatin refers to regions that are devoid of genes or regions where gene transcription is turned off. DNA binding proteins can attach to specific regions of chromatin. These proteins mediate DNA replication, gene expression, or represent structural proteins important in packaging the chromosomes. Histones are structural proteins of chromatin and are the most abundant protein in the nucleus. In fact, the mass of histones in a chromosome is almost equal to that of DNA. Chromosomes contain five types of these small  proteins: H1, H2A, H2B, H3, and H4. There are two of each of latter four histones that form a structure called the octomeric histone core. The H1 histone is larger than the other histones, and performs a structural role separate from the octomeric histone core in organizing DNA within the chromosome.

The octomeric histone core functions as a spool from which DNA is wound two times. Each histone-DNA spool is called a nucleosome. Nucleosomes occur at intervals of every 200 bases pairs of the DNA helix. In photographs taken with the help of powerful microscopes, DNA wrapped around nucleosomes resembles beads (the nucleosome) threaded on a string (the DNA molecule). The DNA that exists between nucleosomes is called linker DNA. Chromosomes can contain some very long stretches of linker DNA. Often, these long linker DNA sequences are the regulatory portions of genes. These regulatory portions switch genes on when certain molecules bind to them.

Nucleosomes are only the most fundamental organizing structure in the chromosome. They are packaged into structures that are 30 nanometers in size and called the chromatin fiber (compared to the 2 nm DNA double helix, and 11 nm histone core). The 30 nanometer fibers are then further folded into a larger chromatin fiber sometimes that is approximately 300 nanometers thick and represent on of the arms of the chromsome. The chromatin fibers are formed into loops by another structural protein. Each loop contains 20,000–30,000 nucleotide pairs. These loops are then arranged within the chromosomes, held in place by more structural proteins. Metaphase chromosomes are approximately 1400 nm wide.

Yogesh Agrawal  &  Sanjoy Gupta

Editor in Chief

Biotech Trove’s Blog

Courtesy  :-   http://science.jrank.org/

PATENTS IN PLANT BREEDING

Posted April 29, 2010 by Sanjoy Gupta
Categories: Science

Guarding the Green Assets

Raj Hirwani,
Is Head, CSIR’s Unit for Research and Development of Information Products, Pune-411 038 (E-mail: hirwani@urdip.res.in)
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Plant variety management relates to the conservation, use and commercial exploitation of plant varieties by farmers, commercial breeders, governments and relevant research organisations. Plant variety protection is one subset of this broad field that focuses exclusively on knowledge, which can be commercially exploited. In other words, plant variety protection relates to intellectual property rights over plant varieties, which guarantee the rights holders’ exclusive commercial rights for a specific period of time.

In the agricultural field, inventiveness was traditionally based on sharing of biological resources and related knowledge among farmers in most parts of the world. This changed gradually in the early part of the 20th century in certain western countries where a private sector seed industry slowly developed. The development of the private sector in this field led to a call for a form of intellectual property rights protection over plant varieties to give sufficient incentive to the private sector to enter the seed business.

Historically, the patent system has been ill adapted to plant varieties. Plant breeders first sought protection under the industrial patent system. However, a number of technical difficulties were encountered in seeking to apply the rules of a system designed to protect technical inventions to plant varieties, which were thought not to precisely reproduce themselves, and whose appearance can vary depending upon the environment in which they are grown. There were two main reasons why the patent system was seen as inappropriate. First, plant material was not regarded as capable of meeting the requirements of novelty, inventive step and disclosure. Second, it was not thought to be in the public interest to permit such an extensive monopoly over plant varieties, given their societal importance. Underlying this was the view that it was desirable to retain, in so far as it was possible, the tradition of free exchange of new plant material between plant breeding institutes. This would ensure the widest possible dissemination and use of the new combinations of genetic information.

However, the scope of patentable subject matter expanded, slowly and incrementally until it covered plants. There has been a move from a strict prohibition against the patenting nature towards a range of court decisions allowing the patenting of living matter. As a result there has been a progressive accommodation of biotechnology within the legal system.
In the United States of America (US) the Supreme Court noted that there is a clear statutory distinction between products of nature and manmade inventions, rather than between living and inanimate things.

US was the first country to promulgate the Plant Patent Act in 1930 that first allowed for patenting of asexually reproduced cultivars (except tubers). By 1960’s some European countries enacted plant breeders’ rights laws. It was demonstrated that sexually reproduced varieties were uniform and stable enough to be included in these laws. US followed Europe by enacting The Plant Variety Protection (PVP) Act in 1970. Its purpose is to “encourage the development of novel varieties of sexually reproduced plants” by providing their owners with exclusive marketing rights in the US.

UNITED STATES
In the US there are three main ways in which an inventor or breeder may obtain formal IPR on plant material:

  • Plant Patent under the Plant Patent Act (PPA),
  • Utility patent, under the Utility Patent Act (UPA), and
  • Plant Breeder’s Rights through the Plant Variety Protection Act (PVPA)

Plant Patents
A plant patent is granted by the US Government to an inventor who has invented or discovered and asexually reproduced a distinct and new variety of plant, other than a tuber propagated plant or a plant found in an uncultivated state. The grant, which lasts for 20 years from the date of filing the application, protects the inventor’s right to exclude others from asexually reproducing, selling, or using the plant so reproduced. This protection is limited to a plant in its ordinary meaning:

  • A living plant organism which expresses a set of characteristics determined by its single, genetic makeup or genotype, which can be duplicated through asexual reproduction, but which can not otherwise be “made” or “manufactured.”
  • Sports, mutants, hybrids, and transformed plants are comprehended (sports or mutants may be spontaneous or induced). Hybrids may be natural, from a planned breeding program, or somatic in source. While natural plant mutants might have naturally occurred, they must have been discovered in a cultivated area.
  • The term “plant” has been interpreted to mean “plant” in the ordinary and accepted sense and not in the strict scientific sense. Algae and macro fungi are regarded as plants, but bacteria are not.

Asexual Reproduction
Asexual reproduction is the propagation of a plant to multiply the plant without the use of genetic seeds to assure an exact genetic copy of the plant being reproduced. Any known method of asexual reproduction, which renders a true genetic copy of the plant, may be employed. Acceptable modes of asexual reproduction would include but may not be limited to Rooting Cuttings, Grafting and Budding, Apomictic Seeds, Bulbs, Division, Slips, Layering, Rhizomes, Runners, Corms, tissue Culture and Nucellar Embryos.

The purpose of asexual reproduction is to establish the stability of the plant. This second step of the invention must be performed well in advance of the application for patent rights, to allow for a thorough evaluation of propagules or clones of the claimed plant for stability. This is to assure that such specimens retain the identical distinguishing characteristics of the original plant.

Other Requirements of Patentability:

  • That the plant was invented or discovered and, if discovered, that the discovery was made in a cultivated area.
  • That the plant is not a plant, which is excluded by statute, where the part of the plant used for asexual reproduction, is not a tuber food part, as with Irish potato or Jerusalem artichoke.
  • That the person or persons filing the application are those who actually invented the claimed plant; i.e., discovered or developed and identified or isolated the plant, or asexually reproduced the plant.
  • That the plant has not been sold or released in the United States of America more than one year prior to the date of the application.
  • That the plant has not been enabled to the public, i.e., by description in a printed publication in the United States more than one year before the application for patent with an offer to sale; or by release or sale of the plant more than one year prior to application for patent.
  • That the plant be shown to differ from known, related plants by at least one distinguishing characteristic, which is more than a difference caused by growing conditions or fertility levels, etc.
  • The invention would not have been obvious to one skilled in the art at the time of invention by applicant.

Due Diligence
Before an application is filed, the (clones of the) plant must have been carefully observed during the testing process. Among the factors which must be ascertained for a reasonably complete botanical description for the claimed plant are:

  • Genus and species
  • Habitat of growth
  • Cultivar’s name
  • Vigor
  • Productivity
  • Precocity (if applicable)
  • Botanical characteristics of plant structures (i.e. buds, bark, foliage, flowers, fruit, etc.)
  • Fertility (Fecundity)

Other characteristics which distinguish the plant such as resistance(s) to disease, drought, cold, dampness, etc., fragrance, coloration, regularity and time of bearing, quantity or quality of extracts, rooting ability, timing or duration of flowering season, etc.

Rights Conveyed by a Plant Patent
A plant patent is granted on the entire plant. It therefore follows that only one claim is necessary and only one is permitted.

Grant of a patent for a plant precludes others from asexually reproducing or selling or using the patented plant. A plant patent is regarded as limited to one plant, or genome. A sport or mutant of a patented plant would not be considered to be of the same genotype, would not be covered by the plant patent to the parent plant, and would, itself, be separately patentable, subject to meeting the requirements of patentability. As with utility applications, when the plant patent expires after 20 years from the filing date of application, the subject matter of the patent becomes public domain. As a part of patent right, Inventor gets an opportunity to give the Distinctive Name to the variety developed by him.

Utility Patents
Utility patents may be granted in the U.S. for any new plant in which man has had “a hand” in the creation thereof. This follows from the landmark rulings in the Diamond vs. Chakrabarty case and also the Ex-parte Hibberd case. US patent law provides that an entity may make claims to a time-limited right to exclude others from use of plants and plant products, provided that the legal criteria for patentability (novelty, non-obviousness and utility) are met. Utility patents may be used to claim exclusionary rights in new varieties of plants , transgenic plants, plant groups, individual plants and their descendants, particular plant traits, plant parts, plant components (e.g. specific genes or chromosomes), plant products (e.g. fruits, oils, pharmaceu-ticals), plant material used in industrial processes (e.g. cell lines used in cultivation methods), reproductive material (e.g. seeds or cuttings), plant culture cells, plant breeding methodologies, and vectors and processes involved in the production of transgenic plants. Note that this list is only illustrative, and not exhaustive. In the utility patent application, the applicant must fully disclose how to identify, make and use the claimed invention.

EUROPEAN PATENT OFFICE
Standard Patents: In countries that are members of the European Patent Office (EPO), the patenting of plant varieties, per se, is prohibited. Indeed, before a decision by the EPO’s Enlarged Board of Appeals on December 20, 1999, it was assumed that no plants could be a subject of a utility patent claim, based on the Directive 98/44/EC of the European Parliament. However, the EPO, Board Of Appeals (BOA) determined that a claim directed to transgenic plants of more than one variety, but that does not claim an individual plant variety, is permissible. Thus, opening the way, for all intents and purposes for the EPO to allow claims to plants.

AUSTRALIA
SAustralia grants two kinds of patents: Standard Patents and Innovation Patents

Standard Patents:
Australia allows the claiming of protection of plants in standard patents, for plants in general and for specific cultivars. The range of patentable subject matter for plants under Standard Patent Applications includes most of the items covered under US Utility Patents.

For inventions relating to plant varieties production include genetic engineering techniques, tissue culture, cell and protoplast culture, mutagenesis and breeding and cultivation methods. A standard patent gives long-term protection and control over an invention for up to 20 years.

Innovation Patents: This relatively fast, inexpensive protection option, lasting a maximum of 8 years was introduced in 2001. Plants and the biological processes for the generation of plants are not patentable subject matter for an innovation patent. However, it is possible to obtain an innovation patent on processes that use a plant or parts of plants, but that does not result in the generation of a plant. In addition to above, Plant Breeders Rights are available for Plant Cultivars only.

JAPAN
Standard Patents: According to the Japanese patent regulations, in an invention relating to a plant, a claim should be described as follows: In the case of an invention of a plant per se, the plant should be specified by, for example, a combination of any of the species, the distinctive gene of the plant, characteristics of the plant, etc. and may be further specified by the process for creating the plant.

KOREA
The Korean Patent Act, which previously limited the scope of patenx-table plant inventions to “a variety of plant that reproduces itself asexually,” has been abolished by recent amend-ments to the Patent Act, which became effective on October 1 2006. As a result, “any new variety of plant” is eligible for protection if it possesses the necessary requirements of a patent application. This makes a sexually reproductive, genetically altered plant also patentable subject matter, regardless of its reprod-uction method, unless it is deemed contrary to public interest.

In addition to the broadening of the patentable subject matter of plant inventions, the Korean Intellectual Property Office (KIPO) has decided to incorporate a deposit system, allowing plants and seeds to be placed in a depository. Thus, making a deposit can more easily satisfy the description requirement of the reproducibility of a plant invention.

NEW ZEALAND
Standard Patents: Plant material, especially transgenic plant varieties, is considered to be patentable Inventions, under the rules for granting utility patents, in New Zealand.

PLANT VARIETY PROTECTION IN OTHER COUNTRIES
With few exceptions, most countries do not permit plant patents. Instead, they offer a protection system that is generally called “plant breeder’s rights.” which include almost all plant types, including sexually and asexually reproducing plants, tubers, and certain hybrids.

INDIA
The World Trade Organisation’s Agreement on Trade-Related Aspects of Intellectual Property Rights (TRIPs) requires member states to provide protection for plant varieties either by patents or by an effective sui generis (stand alone) system, or a combination of the two. The International Union for the Protection of New Varieties of Plants (UPOV) also regulates plant breeders’ rights internationally.

By taking into consideration the requirements under TRIPS and the model provided by UPOV India has enacted The Protection of Plant Varieties and Farmers Rights Act (PPVFR) in 2001. For a variety to be eligible for registration under the above act, it must conform to the criteria of novelty, distinctiveness, uniformity and stability (NDUS), as described below:

  • Novel, if, at the date of filing of the application for registration for protection, the propagating or harvested material of such a variety has not been sold or otherwise disposed of by or with the consent of its breeder or his successor for the purposes of exploitation of such variety (i) in India, earlier than one year, (ii) or outside India, in the case of trees or vines earlier than six years, or, in any other case, earlier than four years, before the date of filing such applications.
  • Distinct, if it is clearly distinguishable by at least one essential characteristic from any other variety whose existence is a matter of common knowledge in any country at the time of filing of the application.
  • Uniform, if subject to the variation that may be expected from the particular features of its propagation, it is sufficiently uniform in its essential characteristics.
  • Stable, if its essential characteristics remain unchanged after repeated propagation or, in the case of a particular cycle of propagation, at the end of each such cycle.

The duration of protection of registered varieties is different for different crops which are as follows: For trees and vines (18 years), For other crop (15 years), For extant varieties (15 years) from the date of notification of that variety.

The Act is progressively being implemented; in the initial phase the Government notified some cereal crops and legumes in order to establish the system of listing of plant varieties for the purpose of registration. Last year two fiber crops were added to the list. The criteria for consideration of priority selection of the crops is the crops on which we are dependent for food and nutritional security, including major cereals, pulses, oilseeds, vegetables and fruits crops. Crop species important for India in the world trade, species of Indian origin, crops where India could benefit from introduction of new germplasm and foreign investment.

As of July 2009 end, the Central Government has notified the following crops eligible for registration of varieties (the figures in the bracket indicates the number of applications received).

Rice (39),Maize(33),Bread Wheat(9),Pigeon pea(6),Pearl Millet (20),Sorghum(36),Chick pea(7),Garden pea(3),French bean(0),Masoor (0),Black gram(6),Green gram (4)Cotton(164)and Jute(5).

Out of total 332 applications, 98 are for new varieties, 225 for extant varieties and 9 for farmer’s varieties.
The guidelines for DUS testing of oilseed crops and few other crops have also been notified.

PROTECTION UNDER THE INDIAN PATENTS ACT
India has amended its patent law in conformity to TRIPS agreement and a new law has come into force from 1st January 2005. Under the amended act, patents will also be granted for biochemical, biotechnological and microbiological processes. This means novel biotechnological processes/methods/techniques employed for the development of new plant varieties including transgenic plants can be patented, but, the plants per se are not patentable.
Yogesh Agrawal  &  Sanjoy Gupta

Editor in Chief
Biotech Trove’s  Blog

Courtesy  :-  http://www.biotechnews.in/

BIOTECHNOLOGY HERITAGE AWARD 2010

Posted April 27, 2010 by Sanjoy Gupta
Categories: Science

Biotechnology Heritage Award – 2010  –  Goes to Arthur D. Levinson



The Chemical Heritage Foundation (CHF) and the Biotechnology Industry Organization (BIO) has selected Arthur D. Levinson as the winner of the 2010 Biotechnology Heritage Award. The award will be presented during BIO’s annual international convention, which will be held in Chicago from 3–6 May 2010.

About Arthur D. Levinson
Arthur D. Levinson is chairman of Genentech. Levinson joined Genentech in 1980 as a research scientist. He became vice president of research technology in 1989, vice president of research in 1990, senior vice president of research in 1992, and senior vice president of R&D in 1993. He served as CEO of Genentech from 1995 to 2009. He has been chairman of Genentech since 1999.

Levinson is a director of Apple and of NGM Biopharmaceuticals. He also serves on the Board of Scientific Consultants of the Memorial Sloan-Kettering Cancer Center, on the Industrial Advisory Board of the California Institute for Quantitative Biomedical Research, on the Advisory Council for the Princeton University Department of Molecular Biology, on the Advisory Council for the Lewis-Sigler Institute for Integrative Genomics, and on the Executive Council of TechNet.

Levinson has authored or coauthored more than 80 scientific articles and has been a named inventor on 11 U.S. patents. He has received numerous awards, including the Irvington Institute’s Corporate Leadership Award in Science, the Corporate Leadership Award from the National Breast Cancer Coalition, and Princeton University’s James Madison Medal for a distinguished career in scientific research and in biotechnology.

Business Week named Levinson one of the Best Managers of the Year in 2004 and 2005, and Institutional Investor named him America’s Best CEO in the biotech category four years in a row (2004–2007). In 2008 Levinson was elected a fellow of the American Academy of Arts and Sciences.

Levinson received a B.S. in molecular biology from the University of Washington and a Ph.D. in biochemical sciences from Princeton University.

Yogesh Agrawal  &  Sanjoy Gupta

Editor in Chief

The Biotech Trove Blog

GENE THERAPY TECHNIQUE

Posted April 26, 2010 by Sanjoy Gupta
Categories: Science

Gene therapy is using “genes as medicine”. It is an experimental approach to treating genetic disease where the faulty gene is fixed, replaced or supplemented with a healthy gene so that it can function normally. Most genetic diseases cannot be treated, but gene therapy research gives some hope to patients and their families as a possible cure. However, this technology does not come without risks and many clinical trials to evaluate its effectiveness need to be done before gene therapy can be put to regular medical use.

How is gene therapy done?

To get a new gene into a cell’s genome, it must be carried in a molecule called a vector. The most common vectors currently being used are viruses, which naturally invade cells and insert their genetic material into that cell’s genome. To use a virus as a vector, the virus’ own genes are removed and replaced with the new gene destined for the cell. When the virus attacks the cell, it will insert the genetic material it carries. A successful transfer will result in the target cell now carrying the new gene that will correct the problem caused by the faulty gene.

Viruses that can be used as vectors include retroviruses like HIV, adenoviruses (one of which causes the common cold), adeno-associated viruses and herpes simplex viruses. There are also many non-viral vectors being tested for gene therapy uses. These include artificial lipid spheres called liposomes, DNA attached to a molecule that will bind to a receptor on the target cell, artificial chromosomes and naked DNA that is not attached to another molecule at all and can be directly inserted into the cell.

The actual transfer of the new gene into the target cell can happen in two ways: ex vivo and in vivo. The ex vivo approach involves transferring the new gene into cells that have been removed from the patient and grown in the laboratory. Once the transfer is complete, the cells are returned to the patient, where they will continue to grow and produce the new gene product. The in vivo approach delivers the vector directly to the patient, where transfer of the new gene will occur in the target cells within the body.

Applications of gene therapy

Conditions or disorders that result from mutations in a single gene are potentially the best candidates for gene therapy. However, the many challenges met by researchers working on gene therapy mean that its application is still limited while the procedure is being perfected.

Before gene therapy can be used to treat a certain genetic condition or disorder, certain requirements need to be met:

  • The faulty gene must be identified and some information about how it results in the condition or disorder must be known so that the vector can be genetically altered for use and the appropriate cell or tissue can be targeted.
  • The gene must also be cloned so that it can be inserted into the vector.
  • Once the gene is transferred into the new cell, its expression (whether it is turned on or off) needs to be controlled.
  • There must be sufficient value in treating the condition or disorder with gene therapy – that is, is there a simpler way to treat it?
  • The balance of the risks and benefits of gene therapy for the condition or disorder must compare favourable to other available therapies.
  • Sufficient data from cell and animal experiments are needed to show that the procedure itself works and is safe.
  • Once the above are met, researchers may be given permission to start clinical trials of the procedure, which is closely monitored by institutional review boards and governmental agencies for safety.

Clinical trials for gene therapy in other countries (for example France and the United Kingdom) have shown that there are still several major factors preventing gene therapy from becoming a routine way to treat genetic conditions and disorders. While the transfer of the new gene into the target cells has worked, it does not seem to have a long-lasting effect. This suggests that patients would have to be treated multiple times to control the condition or disorder. There is also always a risk of a severe immune response, since the immune cells are trained to attack any foreign molecule in the body. Working with viral vectors has proven to be challenging because they are difficult to control and the body immediately recognizes and attacks common viruses. Recent work has focussed on potential non-viral vectors to avoid the complications associated with the viral vectors. Finally, while there are thousands of single-gene disorders, the more common genetic disorders are actually caused by multiple genes, which do not make them good candidates for gene therapy.

One promising application of gene therapy is in treating type I diabetes. Researchers in the United States used an adenovirus as a vector to deliver the gene for hepatocyte growth factor (HGF) to pancreatic islet cells removed from rats. They injected the altered cells into diabetic rats and, within a day, the rats were controlling their blood glucose levels better than the control rats. This model mimics the transplantation of islet cells in humans and shows that the addition of the HGF gene greatly enhances the islet cells’ function and survival.

In Canada, researchers in Edmonton, Alberta also developed a protocol to treat type I diabetes. Doctors use ultrasound to guide a small catheter through the upper abdomen and into the liver. Pancreatic islet cells are then injected through the catheter into the liver. In time, islets are established in the liver and begin releasing insulin.

Another application for gene therapy is in treating X-linked severe combined immunodeficiency (X-SCID), a disease where a baby lacks both T and B cells of the immune system and is vulnerable to infections. The current treatment is bone marrow transplant from a matched sibling, which is not always possible or effective in the long term. Researchers in France and the United Kingdom, knowing the disease was caused by a faulty gene on the X chromosome, treated 14 children by replacing the faulty gene ex vivo. Upon receiving the altered cells, the patients showed great improvements in their immune system functions. Unfortunately, two of the children developed a form of leukemia several years after the treatment. Further investigation showed that the vector had inserted the gene near a proto-oncogene, which led to uncontrolled growth of the T cells. The clinical trials were put on hold until a safer method can be designed and tested.

Sanjoy Gupta

Editor in Chief

Biotech Trove Blog

Courtsey  :-   http://www.hc-sc.gc.ca

Good Smell can Enhance your Lifespan !

Posted April 24, 2010 by Sanjoy Gupta
Categories: Science

In our endeavor to bring to you interesting news from the realm of Biotechnology, we are working hard.  And now an amazing news – it seems that smell of  good meals on a regular basis may increase your lifespan !  Simply read on the following

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What does the smell of a good meal mean to you? It may mean more than you think. Specific odors that represent food or indicate danger are capable of altering an animal’s lifespan and physiological profile by activating a small number of highly specialized sensory neurons, researchers at the University of Michigan, University of Houston, and Baylor College of Medicine have shown in a study in the online, open-access journal PLoS Biology.


Recent research in model organisms and in humans has shown that sensory experiences can impact a wide range of health-related characteristics including athletic performance, type II diabetes, and aging. Nematode worms and fruit flies that were robbed of their ability to smell or taste, for example, lived substantially longer. However, the specific odors and sensory receptors that control this effect on aging were unknown.

Using molecular genetics in combination with behavioral and environmental manipulations, a collaboration between the laboratories of Scott Pletcher and Gregg Roman has succeeded in identifying carbon dioxide (CO2) as the first well-defined odorant capable of altering physiology and affecting aging. Flies incapable of smelling CO2 live longer than flies with normal olfactory capabilities. They are also resistant to stress and have increased body fat. To many insects, including fruit flies, CO2 represents an ecologically important odor cue that indicates the presence of food (e.g. rotting fruit or animal blood) or neighbors in distress (it has been implicated as a stress pheromone). Indeed, this group of researchers previously showed that merely sensing one’s normal food source is capable of reversing the health and longevity benefits that are associated with a low calorie diet. They now establish that CO2 is responsible for this effect.

“We are working hard to understand how sensory perception affects health, and our new result really narrows the playing field. Somehow these 50 or so neurons, whose primary job it is to sense CO2, are capable of instigating changes that accelerate aging throughout the organism,” says Scott Pletcher.

Sensory perception has been shown to impact aging in species that are separated by millions of years of evolution, suggesting that similar effects may be seen in humans. “For us, it may not be the smell of yeast, for example, or the sensing of CO2 that affects how long we live, but it may be the perception of food or danger,” says Pletcher. If so, a clever program of controlled perceptual experience might form the basis of a simple yet powerful program of disease prevention and healthy aging.

Sanjoy Gupta

The Editor in Chief

The Biotech Trove’s Blog


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