Open Questions: Gene Therapy

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See also: Drug delivery -- Nanobiotechnology -- RNA biology -- Gene expression and regulation

Introduction


Recommended references: Web sites

Recommended references: Magazine/journal articles

Recommended references: Books

Introduction



Recommended references: Web sites

Site indexes

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 found at Netscape.
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 external links.


Surveys, overviews, tutorials

Gene Therapy
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 Web site.
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 additional resources.
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."
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.
Gene Therapy
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.


Recommended references: Magazine/journal articles

The Second Coming of Gene Therapy
Jill Neimark
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
Jocelyn Kaiser
Smithsonian, January 2009
Delivering the Goods
Jessica Gorman
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
Jeff Wheelwright
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 gene therapy.
[Abstract and references]
Infectious Notion
Ruth Bennett
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
Ken Garber
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.
Gene Therapy
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
Theodore Friedmann
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.


Recommended references: Books


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