Scientists at Emory University School of Medicine have discovered that drugs that bind dissolved iron, called iron chelators, can enhance RNA interference.

Maternal Genetic Mutation and Use of Folic Acid Supplements May Increase Risk for Childhood Eye Cancer

Gene therapy for hearing loss: potential and limitations

Regenerating sensory hair cells, which produce electrical signals in response to vibrations within the inner ear, could form the basis for treating age- or trauma-related hearing loss. One way to do this could be with gene therapy that drives new sensory hair cells to grow.

Secret of sperm quality control revealed by Yale scientists

By Bill Hathaway

Yale researchers have discovered how the “guardian of the genome’’ oversees quality control in the production of sperm — and perhaps in many other cells as well.

Yale study proves nobody is genetically perfect

By Bill Hathaway

Every person carries on average 100 variants that disable genes - yet very few suffer ill effects, an international team of researchers led by Yale University and Wellcome Trust Sanger Institute report in the Feb. 17 issue of the journal Science.

Caltech Researchers Develop Gene Therapy to Boost Brain Repair for Demyelinating Diseases

PASADENA, Calif.—Our bodies are full of tiny superheroes—antibodies that fight foreign invaders, cells that regenerate, and structures that ensure our systems run smoothly. One such structure is myelin—a material that forms a protective, insulating cape around the axons of our nerve cells so that they can send signals quickly and efficiently. But myelin, and the specialized cells called oligodendrocytes that make it, become damaged in demyelinating diseases like multiple sclerosis (MS), leaving neurons without their myelin sheaths. As a consequence, the affected neurons can no longer communicate correctly and are prone to damage. Researchers from the California Institute of Technology (Caltech) now believe they have found a way to help the brain replace damaged oligodendrocytes and myelin.

	Caltech researchers promote repair in a mouse model of MS by enhancing the production of myelin producing cells (shown in green). Credit: Benjamin Deverman/Caltech

Gene therapy for inherited blindness succeeds in patients' other eye

In 3 Adults, Repeat Dose Safely Improves Vision

PHILADELPHIA – Gene therapy for congenital blindness has taken another step forward, as researchers further improved vision in three adult patients previously treated in one eye. After receiving the same treatment in their other eye, the patients became better able to see in dim light, and two were able to navigate obstacles in low-light situations. No adverse effects occurred.

Gene linked to pancreatic cancer growth, U-M study finds

ANN ARBOR, Michigan.— Una proteína mutante que se encuentra en casi todos los cánceres de páncreas desempeña un papel no sólo en el desarrollo del cáncer sino también en su crecimiento continuo, según un nuevo estudio de los investigadores en el Centro Integral del Cáncer de la Universidad de Michigan. Este descubrimiento indica un posible enfoque para el desarrollo de nuevas modalidades en el tratamiento de esta enfermedad mortal.

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Gene Therapy Research Team From Penn Vet and Scheie Eye Institute Cures Retinitis Pigmentosa in Dogs

PHILADELPHIA -- Members of a University of Pennsylvania research team have shown that they can prevent, or even reverse, a blinding retinal disease, X-linked Retinitis Pigmentosa, or XLRP, in dogs.

Survey Reveals Scientists Have Trouble Accessing Human Embryonic Stem Cell Lines

The promise of stem cell research for drug discovery and cell-based therapies depends on the ability of scientists to acquire stem cell lines for their research.

Columbia Researchers Pinpoint Fault in Breast Cancer Gene

In a study published October 28, 2011, in Science, researchers from the Institute for Cancer Genetics and Herbert Irving Comprehensive Cancer Center have finally pinpointed the flaw in BRCA1 that causes cancer.

Vast Hidden Network Regulates Gene Expression in Cancer

Researchers at Columbia University Medical Center (CUMC) and two other institutions have uncovered a vast new gene regulatory network in mammalian cells that could explain genetic variability in cancer and other diseases. The studies appear in today’s online edition of Cell.

Shutting off inflammation

Image: wikipedia/Vossman

A Gene That Fights Cancer, But Causes It Too

Over-activation of a single gene promotes leukemia, but its loss causes liver cancer

A New Way to Make Reprogrammed Stem Cells

Penn study eliminates the use of transcription factors and increases efficiency 100-fold

PHILADELPHIA - Researchers at the University of Pennsylvania School of Medicine have devised a totally new and far more efficient way of generating induced pluripotent stem cells (iPSCs), immature cells that are able to develop into several different types of cells or tissues in the body. The researchers used fibroblast cells, which are easily obtained from skin biopsies, and could be used to generate patient-specific iPSCs for drug screening and tissue regeneration.

NIH researchers extend use of gene therapy to treat a soft tissue tumor

Potential cancer-causing genes removed from engineered stem cells
Whitehead work involved reprogrammed skin cells from Parkinson's patient

RNA interference can suppress ovarian tumor growth
Study offers promise for new cancer treatments

Multiple genes implicated in autism
Discovery could lead to drugs targeting gene interactions