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Links to Gene Therapy

Primers on DNA & Genomics

An animated primer on the basics of DNA, genes, and heredity.  Includes animation, image gallery, video interviews, problems, biographies, and links. Produced by DNA Learning Center, Cold Spring Harbor Laboratory.

Explanation of DNA, chromosomes, cell constituents, and genetic disorders. Produced by University of Utah, Genetic Science Learning Center.

Excellent primer on human genomics.

Excellent background material on genes and genomics.  Accessed through this site as either html or pdf file.

A solid primer overview of DNA, genomics, gene testing, and genetic research.  Access through this site as either html or pdf file.

(from Human Genome Project)

Primers on Gene Therapy

Excellent primer.

Excellent review article on human gene therapy from "The Father of Gene Therapy".

Outstanding, classic review article on gene therapy from "The Father of Gene Therapy".

(September 2000)

Frequently Asked Questions (FAQs) About Gene Therapy

(from the UCLA Program on Medicine Technology and Society)

Gene Therapy Trial Sources

A comprehensive listing of human gene therapy clinical trials in the U.S.  All studies are in "somatic" gene therapy.

An excellent interactive source for human gene therapy clinical trials conducted around the world.

U.S. Research Centers




"The UI College of Medicine Gene Transfer Vector Core brings researchers interested in developing and using gene transfer together with specialists in the area of viral vector construction.  The core utilizes molecular biology techniques to engineer and produce viral and non-viral vectors in quantities necessary for gene transfer in research experiments or preclinical studies. Core staff work closely with investigators to plan and develop gene transfer vectors to fit individual project requirements."


"The Center facilitates the understanding of genetic mechanisms, the development of genetically based technology for diagnosis and treatment of disease and aids the faculty of Northwestern University's Feinberg School of Medicine and its partner hospitals in the application of genetics to the practice of medicine."






North Carolina

"Research in the Gene Therapy Center at the University of North Carolina has centered on the study of the molecular biology of adeno-associated virus (AAV) in order to exploit the unique features of this virus to develop an efficient viral vector system for use in human gene therapy. The core facilities available to gene therapy investigators include the Vector Core Laboratory and the Human Applications Laboratory (a GMP facility). These facilities were created to ensure that investigators would have promising gene vectors available in the quality and quantities needed for pre-clinical and clinical studies."

New York


"The mission of the Molecular Medicine Institute is to serve as a catalyst for the growth and development of multidisciplinary pre-clinical and clinical research programs aimed at the use of novel molecular therapies, including cell, protein and gene therapeutic methods, for the treatment of human disease.  In order to fulfill our mission, the following objectives have been outlined: (1) provide state-of-the-art core facilities and broad-based expertise in gene therapy to both the University community and research community, at large; (2) provide the resources and infrastructure to facilitate compliance with existing regulations and to stay abreast of new regulatory issues related to vector development and manufacturing, as well as, clinical trial management and oversight; (3) provide assistance with clinical protocol design and application to the various clinical regulatory entities include the RAC, IBC, IRB, and FDA to enable new Phase I/Phase II clinical trials in the field of molecular medicine to be initiated in a timely manner; (4) foster collaboration between scientists and clinicans to encourage creatvitiy and innovation in the science, technology, and clinical practice of molecular medicine; and (5) train future generations of scientists and clinicians in the field of molecular medicine."


"The Center for Cell and Gene Therapy will interact with Basic Research departments at Baylor College of Medicine and its affiliated hospitals to discover novel therapies for a range of diseases. It is our intention to have the capability of moving basic pre-clinical discoveries into the clinical phase of development for initial evaluation in our patients. To allow this, we will have the resources to manufacture a wide range of viral and non-viral vectors to transfer genes of interest and the capability of separating and manipulating human cells. These combined genetic and cellular therapies should enable us to treat a range of conditions, both congenital and acquired, in almost every system of the body."


Professional Societies for Gene Therapy or Genetics in North America

United States


Professional Societies for Gene Therapy or Genetics in Europe

Europe (Overall)

"European Society of Gene Therapy (ESGT): a society dedicated to the development of gene therapies, cell therapies and genetic vaccines. The primary objectives of ESGT are to promote basic and clinical research in gene and cell therapy, to promote education and the exchange of information and technology related to gene transfer and therapy, and to serve as a professional adviser to the community and to the regulatory bodies in Europe."

"ESHG is an international professional society founded in 1967 which promotes research in basic and applied human and medical genetics and facilitates contact between all persons who share these aims."

United Kingdom

"BSHG is an independent body representing UK human genetics professionals, working in all areas from cutting edge research to the delivery of genetic services to the population of the UK."

Europe (Continent)

Professional Societies for Gene Therapy or Genetics in Australia

Organizations Involved in Ethics Discussions Concerning (Germline) Gene Therapy and/or Genetic Engineering


"It is the mission of the Center for the Study of Technology and Society to research and analyze the interaction of technological change and society.  The Center is a nonprofit think tank based in Washington, D.C."

"The Council for Responsible Genetics fosters public debate about the social, ethical and environmental implications of genetic technologies.  Founded in 1983, CRG is a non-profit, non-governmental organization based in Cambridge, massachusetts. CRG works through the media and concerned citizens to distribute accurate information and represent the public interest on emerging issues in biotechnology. CRG also publishes a bimonthly magazine, GeneWatch, the only publication of its kind in the nation."

"GeneWatch UK is a public interest group which aims to ensure that genetic technologies are developed and used in the public interest and in a way which promotes human health, protects the environment and respects human rights and the interests of animals."

"GEENOR---the Genetic Engineering Organziation--is a private collective of people who are unanimously in favor of genetic engineering. GEENOR is established to foster genetic engineering as the best way for humanity to progress."

"Geneforum is an Oregon based nonprofit dedicated to promoting civic discourse about social and ethical issues arising in the new world of genetic science and technology. In that role, we help the general populace learn more about genetic science aimed at creating an informed and engaged citizenry with the capacity to help guide the development of genetic policies which reflect public values."

"The Genetics Forum is a public interest group exclusively devoted to policy development, campaigns and publications on genetic engineering from a social, ethical and environmental perspective."

"Ifgene---International Forum for Genetic Engineering---provides opportunities for power-free dialogue in a context of pluralism. It tries to enhance the process of forming judgements about aspects of genetic engineering by enquiring into the presuppositions behind the viewpoints, including scientific, that are presented. It does not confine the dialogue to one or a narrow spectrum of world views.  It aims to raise public awareness without becoming partisan."

Academic Centers for Bioethics

  • Human Germline Engineering,

Ethics Megalinks

Extensive listing of bioethics resources at NHGRI, National Institutes of Health, U.S. Dept of Health & Human Resources (DHHS), U.S. Dept. of Energy (DOE), and other resources

"The National Human Genome Research Institute's (NHGRI) Ethical, Legal and Social Implications (ELSI) Program was established in 1990 as an integral part of the Human Genome Project (HGP) to foster basic and applied research, and support education and outreach.  The ELSI program funds and manages studies related to the ethical, legal and social implications of genetic and genomic research, and supports workshops, research consortia and policy conferences related to these topics.  The ELSI program at NHGRI is the largest supporter nationwide of ELSI research." (Text from NHGRI website.)

Search the National Library of Medicine's PubMed database for articles on bioethics. To narrow the field to bioethics publications, use the drop-down box listed as "subsets" and then choose "bioethics".

Update/News Sources

Sources for Developments in Gene Therapy & Genetics

Sources for Developments in Biotechnology

Hubs + Meta-links

Joint Site of the Department of Energy (DOE), Office of Biological & Environmental Research &
Human Genome Project

Vast connections for exploring genes and genetic disorders including tools and tutorials for evaluating genes and chromosome structures

Detailed links for the human genome as well as database information

MegaSource for genetic links

Main site for human genome information

Key links via Human Genome Project

General Medical Resources

General Medical Resources for Patients and the Public

Key government resource for health and disease topic information

Key government source for information on thousands of prescription and over-the-counter medications

Excellent source for general medical information

Key government source for finding reliable healthcare information

Commercial Website General Medical Resources

Medical Dictionaries

Includes more than 4000 articles about diseases, tests, symptoms, injuries, and surgeries as well as an extensive library of medical photographs and illustrations.  (Taken from MedlinePlus)

Dictionary containing more than 8300 terms relating to biochemistry, biotechnology, botany, cell biology, and genetics.  (From the University of Texas Institute for Cellular and Molecular Biology)

Classic, extensive medical dictionary

Key Databases on Gene Therapy and Genetics

The Key Search Tool for literature holdings of the National Library of Medicine (NLM). PubMed provides access to more than 12 million journal publications/citations in the NLM database (MEDLINE) dating back to the mid-1960s. Most have accompanying abstracts; some provide links to full text articles.

Links to a vast reservoir of information in the National Library of Medicine (NLM) and the National Institutes of Health (NIH) providing information for the public, patients, and physicians on various health topics, drug information , dictionaries, directories, curren health news, and other resources.

Government institue leading the Human Genome Project for NIH, conducting cutting edge research and supporting genomic science worldwide.

Access to human gene mapping results, chromosome by chromosome, section by section, gene by gene. Key for viewing human chromosomes.

Access to current developments in genome sequencing progress as well as access to databases such as BLAST (Basic Local Alignment Search Tool for identifying similarities between different sequences of DNA or proteins).

Catalog of human genes and disease.

Relational database of rare genetic diseases.

Links in functional genomics, publications, analysis of protein and/or DNA sequences.

(Primarily intended for physicians and researchers) Site designed to provide comprehensive links to reliable information about genes, their associated proteins, and genetic mutations associated with cancer and related disorders.

U.S. Government Health Research Agencies


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