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
background material on genes and genomics. Accessed
through this site as either html or pdf file.
solid primer overview of DNA, genomics, gene
testing, and genetic research. Access through
this site as either html or pdf file.
(from Human Genome
Primers on Gene Therapy
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".
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
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
"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
"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."
"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
Professional Societies for Gene Therapy or Genetics
"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."
"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."
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
"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
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
"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".
Sources for Developments in Gene Therapy & Genetics
Sources for Developments in Biotechnology
Hubs + Meta-links
Joint Site of the Department of Energy
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
Detailed links for the human genome as well as database
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
Commercial Website General Medical Resources
Includes more than 4000 articles about diseases, tests,
symptoms, injuries, and surgeries as well as an extensive
library of medical photographs and illustrations. (Taken
Dictionary containing more than 8300 terms relating to
biochemistry, biotechnology, botany, cell biology, and
the University of Texas Institute for Cellular and Molecular
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
Government institue leading the Human Genome Project for
NIH, conducting cutting edge research and supporting genomic
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
U.S. Government Health Research Agencies
- National Center for Biotechnology
Information (NCBI)---The Key (central) source
for searching databases. NCBI creates public databases,
conducts research in computational biology, develops
software tools for analyzing genome data, and disseminates
biomedical information for the better understanding of
molecular processes affecting human health and disease.
to vast array of databases and search tools including genetic,
molecular, literature, retroviral, and sequencing and genome
databases, and search tools such as PubMed, Entrez, nucleotide
and protein sequence analysis, and gene maps.
- National Human Genome
Research Institute (NHGRI)---Key research center
for genetic research.
- NHGRI Genome Hub--Center
for web links about the human genome sequence, projects
to sequence the genomes of other organisms and additional
- Human Genome Project (HGP)--Many
links to information pertaining to sequencing the entire
human genome (DNA structure).
- HGP Educational Resources--For
students, teachers, and the general public.
- Office of Rare Diseases,
National Institutes of Health---Wealth of information
on more than 7000 rare diseases, including current
research, publications from scientific and medical
journals, completed research, ongoing studies, and
patient support groups.
- Food and Drug Administration
(FDA)--Agency responsible for protecting
public health by helping safe and effective pharmaceutical
and medical device products reach the marketplace
in a timely fashion.
for Biologics Evaluation & Research
(CBER)--FDA division which regulates biological
products and ensures their safety for the public.
- Centers for Disease Control & Prevention,
Office of Genomics & Disease Prevention---Provides
information about human genetic discoveries and how they
can be used to improve health and prevent disease.