Gene Therapy News and Research

RSS

Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

Gene therapy can effectively treat ADA-deficient severe combined immunodeficiency

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response.

25 May 2012

Optogenetic Therapies for Vision workshop to be held on June 1

The Foundation Fighting Blindness and Massachusetts Eye and Ear will host the Optogenetic Therapies for Vision workshop on Friday, June 1, from 8 a.m.-6 p.m., in Boston.

24 May 2012

Molecular Therapy releases characteristics and activity of SNS01 formulation

Senesco Technologies, Inc., announced today that the characteristics and activity of SNS01 have been released on-line in Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

24 May 2012

Study shows how chromosomal mutation likely contributes to early development of prostate cancer

Up to half of all prostate cancer cells have a chromosomal rearrangement that results in a new "fusion" gene and formation of its unique protein -- but no one has known how that alteration promotes cancer growth. Now, Weill Cornell Medical College researchers have found that in these cancer cells, the 3-D architecture of DNA, wrapped up in a little ball known as a chromatin, is warped in such a way that a switch has been thrown on thousands of genes, turning them on or off to promote abnormal, unchecked growth. Researchers also found that new chromosomal translocations form, further destabilizing the genome.

23 May 2012

IntelliCell enters sponsored research agreement with ICERM

IntelliCell BioSciences, Inc. announced today that it has entered a sponsored research agreement with the Institute for Cell Engineering and Regenerative Medicine (ICERM) at the University of Florida.

23 May 2012

TransCytos, Enginasion collaborate to develop new transfection technology

Enginasion and TransCytos announced today that their collaboration has resulted in a prototype 'transfection' technology that is designed to have a dramatic and positive impact on the drug-research industry.

22 May 2012

Study uncovers distinct subtype of prostate cancer

A collaborative expedition into the deep genetics of prostate cancer has uncovered a distinct subtype of the disease, one that appears to account for up to 15 percent of all cases, say researchers at Weill Cornell Medical College, the Broad Institute of MIT and Harvard and the Dana-Farber Cancer Institute.

21 May 2012

UK-led technologies set to transform treatments on the NHS

Video games, pioneering gene therapies and new medical devices are set to transform treatments on the NHS, with support from the Department of Health and the Wellcome Trust.

19 May 2012

Promising updates in quest for early detection and treatments for lung cancer

LUNGevity Foundation, the nation's largest lung cancer-focused nonprofit, is pleased to note promising updates in researchers' quest for early detection and treatments for lung cancer during May, Lung Cancer Hope Month.

18 May 2012

Researchers identify novel base modification in RNA

Over the past decade, research in the field of epigenetics has revealed that chemically modified bases are abundant components of the human genome and has forced us to abandon the notion we've had since high school genetics that DNA consists of only four bases.

18 May 2012

Cardiac ischemia plays an important role in adenovector gene transfection in mammalian hearts

Cardium Therapeutics today announced a late-breaking poster presentation at the American Society of Gene & Cell Therapy (ASGCT) 15th Annual Meeting being held May 16-19, 2012 at the Pennsylvania Convention Center in Philadelphia, PA.

17 May 2012

Mary Ann Liebert releases BioResearch Open Access journal

The inaugural issue of BioResearch Open Access, a new bimonthly peer-reviewed open access journal, was released today by Mary Ann Liebert, Inc., publishers. The Journal provides a new rapid-publication forum for a broad range of scientific topics including but not limited to molecular and cellular biology, tissue engineering and biomaterials, regenerative medicine, stem cells, gene therapy, systems biology, genetics, biochemistry, virology, microbiology, and neuroscience.

17 May 2012

Gene therapy restores movement in children bedridden with AADC

Using gene transfer techniques pioneered by University of Florida faculty, Taiwanese doctors have restored some movement in four children bedridden with a rare, life-threatening neurological disease.

17 May 2012

First patient included in the phase II study of Amphinex® in head and neck cancer patients

PCI Biotech (PCIB) the Norwegian biopharmaceutical company, reported today that the first patient has been included in the phase II study of Amphinex in combination with the cytotoxic agent bleomycin in head and neck cancer patients, called the ENHANCE study. The first patient was included at the National Center for Tumor Diseases (NCT), University Hospital Heidelberg, Germany. NCT is a joint project of the German Cancer Research Center (DKFZ), Heidelberg University Hospital, Thoraxklinik Heidelberg and Deutsche Krebshilfe (German Cancer Aid).

16 May 2012

Groundbreaking biohybrid device to control acute inflammation to prevent sepsis

The body's natural inflammatory response is an essential reaction to injury and infection. When acute inflammation escalates out of control, such as in sepsis, it causes nearly 10% of deaths in the U.S. and more than $17 billion in healthcare costs each year. A group of researchers have developed a groundbreaking biohybrid device that can control acute inflammation to prevent sepsis and other related life-threatening complications, as described in an article in the inaugural issue of Disruptive Science and Technology.

14 May 2012

Regenerating sensory hair cells could help treat hearing loss

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.

14 May 2012

Human Gene Therapy highlights new advances in human retinal disease

Gene therapy strategies to prevent and treat inherited diseases of the retina that can cause blindness have progressed rapidly. Positive results in animal models of human retinal disease continue to emerge, as reported in several articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

9 May 2012

Molecule that starts DNA repairs also halts them

Repairing DNA breaks can save a cell's life - but shutting off the repair machinery can be just as critical. How cells accomplish this feat was unknown. However, new research by Johns Hopkins scientists, published in the Feb. 22 issue of Nature, suggests that shutting down the repair machinery relies heavily on the same molecule used to start repair in the first place.

8 May 2012

Automated way to record electrical activity inside neurons in the living brain

Gaining access to the inner workings of a neuron in the living brain offers a wealth of useful information: its patterns of electrical activity, its shape, even a profile of which genes are turned on at a given moment. However, achieving this entry is such a painstaking task that it is considered an art form; it is so difficult to learn that only a small number of labs in the world practice it.

7 May 2012

Scientists identify first gene associated with congenital asplenia

Researchers at Weill Cornell Medical College and Rockefeller University have identified the first gene to be linked to a rare condition in which babies are born without a spleen, putting those children at risk of dying from infections they cannot defend themselves against. The gene, Nkx2.5, was shown to regulate genesis of the spleen during early development in mice.

4 May 2012