A large proportion of genetically caused deafness in humans may be reversible by compensating for a missing protein, based on discoveries in mice.

Carbon nanotubes transport gene therapy drug into T-cells known to block HIV from entering cells in vitro

Three years ago Mark Kay, MD, PhD, published the first results showing that a biological phenomenon called RNA interference could be an effective gene therapy technique. Since then he has used RNAi gene therapy to effectively shut down the viruses that cause hepatitis and HIV in mice.

Scientists at the Center for Translational Medicine at Thomas Jefferson University in Philadelphia have staved off heart failure in animals by using gene therapy to shut down the adrenal gland's excessive output of fight or flight hormones such as epinephrine and norepinephrine. By blocking GRK2, an important regulatory enzyme, they cut the hormone production that forces the heart to pump too hard, leading to heart failure.

Combination gene therapy delivered in lipid-based nanoparticles drastically reduces the number and size of human non-small cell lung cancer tumors in mice, researchers at The University of Texas M. D. Anderson Cancer Center and the University of Texas Southwestern Medical Center report in the Jan. 15 edition of Cancer Research.

Dozens of medical experts from National Cancer Institute, National Institute of Health, FDA, Genentech, Stanford University and others will discuss nanotechnology's promise for preventing, diagnosing, and treating cancer at the CancerNano 2007, May 20-24, 2007 in Santa Clara, Calif.

Since the late 1980s, gene therapy, more than virtually any other type of therapy, has given rise not only to high expectations of treatment success but also great concerns regarding health risks.

The first human trial of gene transfer therapy for erectile dysfunction (ED) indicates that gene therapy that lasts for months and eliminates the patients need for on-demand drugs (such as Viagra and Cialis), could become the future treatment of choice for this common problem, according to a paper in the most recent issue of Human Gene Therapy.