Open Questions: Gene Therapy
See also: Drug delivery --
RNA biology --
Gene expression and regulation
Open Directory Project: Gene Therapy
- Categorized and annotated links. A version of this
list is at
Google, with entries sorted in "page rank" order. May also be
Yahoo News Full Coverage: Gene Therapy
- Links to recent news stories from various sources. Also includes
links to sites dealing with gene therapy.
Sites with general resources
Human Genome Project Information: Gene Therapy
- Good collection of general information on gene therapy.
Includes articles and external links.
Part of the
Human Genome Project Web site.
Institute for Human Gene Therapy
- Contains general information on gene therapy, including
external links, news, a tutorial:
What Is Gene Therapy?, and a more extensive guide to the
basic science of gene therapy.
Cellular & Gene Therapy
- A page with information on some topics in gene therapy, provided
by the U. S.
Food and Drug Administration.
Institute for Genetic Medicine
- Research organization at the University of Southern California.
Page includes a brief overview of gene therapy.
American Society for Gene Therapy
- Professional organization for research in gene therapy.
The site has a number of
Surveys, overviews, tutorials
- Good overview information, with external links, provided
by the U. S.
Human Genome Project.
Questions and Answers About Gene Therapy
- A good FAQ list, at the
National Cancer Institute
Gene Therapy: Molecular Bandage?
- Multi-page tutorial presentation on gene therapy, covering
topics such as choosing targets, gene delivery, tool, and
new experimental approaches. There are links to
Take two genes and call me
- Very elementary overview of gene therapy.
Introduction to Gene Therapy
- Material from a course at Vanderbilt University.
How does gene therapy work?
- May 2008 Scientific American "Ask the Experts" article.
Answer by Arthur Nienhuis.
Regaining Lost Luster
- January 2008 Scientific American article.
"New developments and clinical trials breathe life back into
Gene Therapy for Broken Hearts
- December 2000 Scientific American news article about
a way to deliver a gene for an inhibitory G protein to remediate
abnormal heart rhythms.
- October 1996 Scientific American In Focus article,
subtitled "Early trials encountered unforeseen complications. A new
round of more sophisticated strategies may turn the tide."
Answers emerge on fatal gene therapy trial
- January 2001 news article about possible causes of patient
death in adenovirus gene therapy trial.
Gene therapy cures blindness in dogs
- May 2001 news article in Science News about a gene
therapy test that cured congenital blindness in three dogs.
Posse heads tumour cells off at the pass
- November 2000 news article about an experimental gene therapy
for brain cancer.
Viral vs. Nonviral in Gene Therapy: Which Vector Will Prevail?
- June 1998 news article in The Scientist, about the
trade-offs of using viral or non-viral vectors for gene therapy.
If the pancreas won't, the liver might
- August 1999 news article about experimental genetic modification
of liver cells to produce insulin.
Gene therapy brings haemophilia cure one step closer
- January 1999 news article about an experimental gene therapy
trial for hemophillia in dogs.
The Second Coming of Gene Therapy
Discover, September 2009
- For years, gene therapy has been falling short of meeting
expectations. Recently, though, new approaches have yielded its
first successes: breakthrough treatments for blindness, cancer,
and severe combined immunodeficiency.
Gene Therapy in a New Light
Smithsonian, January 2009
Delivering the Goods
Science News, January 18, 2003, pp. 43-44
- The trick in gene therapy is getting the DNA with the
therapeutic genes into the target cells. Viruses can do this
but have safety problems. Other approaches are being studied,
such as "naked DNA" and artificial containers to transfer DNA.
- Body, Cure Thyself
Discover, March 2002, pp. 62-69
- Gene therapy, theoretically, should be a promising therapeutic
technique for diseases which can be traced to specific genetic
abnormalities, such as severe comibined immune deficiency (SCID).
Yet in spite of much experimentation, success is still elusive.
- Gene Therapy for Pain
Jay Yang; Christopher L. Wu
American Scientist, March-April 2001, pp. 126-135
- Pain can originate from many sources within the body, but
specialized neurons known as "nociceptors" are usually involved.
Various chemical signals are ultimately implicated, and these
may susceptible to modulation within the cell by the use of
[Abstract and references]
Science News, August 19, 2000, pp. 126-127
- Experience with gene therapy guides research in the use of
viruses to treat cancer.
- High Stakes for Gene Therapy
Technology Review, March/April 2000, pp. 58-64
- The ideas behind using DNA as a therapeutic tool are fairly
simple, but practical implementation has proven difficult.
Eric B. Kmiec
American Scientist, May-June 1999, pp. 240-247
- Gene therapy research has tended to focus on adding corrective
genes to cells that contain defective genes. An alternative approach
is to find ways to correct the defects.
- Overcoming the Obstacles to Gene Therapy
Scientific American, June 1997, pp. 96-101
- Introduction to a series of articles on how gene therapy
may be made practical.
- Nonviral Strategies for Gene Therapy
Philip L. Felgner
Scientific American, June 1997, pp. 102-106
- Use of viruses as a delivery mechanism for DNA in gene therapy
poses various problems. Alternatives delivery mechanisms involving
"naked DNA" may be more successful in therapies and vaccines.
- Gene Therapy for Cancer
R. Michael Blaese
Scientific American, June 1997, pp. 111-115
- Use of gene therapy to treat cancer is still in early stages
of investigation. A variety of forms of therapy are being studied.
- Gene Therapy for the Nervous System
Dora H. Yo; Robert M. Sapolsky
Scientific American, June 1997, pp. 116-120
- Several highly debilitating diseases of the nervous system
such as Alzheimer's disease and Parkinson's disease may be amenable
to gene therapy. Introducing appropriate DNA molecules into the
nervous system is especially difficult, but various approaches using
viral vectors are under study.
Copyright © 2002 by Charles Daney, All Rights Reserved