Gene Therapy News and Research

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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.

Johns Hopkins researchers successfully deliver new nanoparticle gene therapy to treat glioma in rats

Despite improvements in the past few decades with surgery, chemotherapy and radiation therapy, a predictably curative treatment for glioma does not yet exist. New insights into specific gene mutations that arise in this often deadly form of brain cancer have pointed to the potential of gene therapy, but it's very difficult to effectively deliver toxic or missing genes to cancer cells in the brain.

5 Feb 2015

Morphogenesis, University of Florida partner to develop novel device to isolate rare cells

Senior engineering students and faculty at the University of Florida in Gainesville are partnering with biotech cell and gene therapy company Morphogenesis, Inc. in Tampa to design an advanced, fully automated cell separation system capable of capturing rare cells – such as stem cells – and circulating tumor cells.

4 Feb 2015

Cell-penetrating nanoparticles can efficiently transport oligonucleotide drugs into cells

Therapeutic oligonucleotide analogs represent a new and promising family of drugs that act on nucleic acid targets such as RNA or DNA; however, their effectiveness has been limited due to difficulty crossing the cell membrane.

31 Jan 2015

Using antioxidant to reverse brain inflammation improves obesity, diabetes symptoms

Using an antioxidant to reverse inflammation in the brain caused by a high-fat diet greatly improves symptoms related to obesity and type II diabetes, a new study from New Zealand's University of Otago suggests.

30 Jan 2015

UCSD professor wins 2015 Japan Prize

Theodore Friedmann, MD, professor in the Department of Pediatrics at University of California, San Diego School of Medicine was named today one of three recipients of the 2015 Japan Prize, a prestigious international award honoring laureates whose "original and outstanding achievements in science and technology have advanced the frontiers of knowledge and served the cause of peace and prosperity for mankind.

29 Jan 2015

TxCell to highlight success factors in cell therapy research at Cell & Gene Therapy Forum 2015

TxCell SA, a biotechnology company developing innovative, cost-effective, personalized T cell immunotherapies using antigen specific regulatory T-cells (Ag-Tregs) for severe chronic inflammatory and autoimmune diseases, announces today that Dr. Miguel Forte, Sr. VP Clinical Development and Regulatory Affairs, TxCell, will present at the Cell & Gene Therapy Forum 2015, January 26 - 28, 2015 at Grand Hayatt in Washington DC, United States.

23 Jan 2015

New Baylor research explores novel non-invasive technology for pediatric eye exams

Picture a toddler getting his first eye exam. He's seated in a strange room, with strange instruments and strange bright lights. He can't sit still or open his eyes long enough for that diagnostic poof of air - especially if he has trouble seeing anyway, as children with achromatopsia do.

23 Jan 2015

Umbilical cord-derived stem cells from women with gestational diabetes show premature aging

Multipotent cells isolated from the human umbilical cord, called mesenchymal stromal cells (hUC-MSCs) have shown promise for use in cell therapy to treat a variety of human diseases. However, intriguing new evidence shows that hUC-MSCs isolated from women with gestational diabetes demonstrate premature aging, poorer cell growth, and altered metabolic function, as reported in an article in Stem Cells and Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

22 Jan 2015

Penn, Spark Therapeutics expand strategic relationship to develop treatment for progressive blindness

The University of Pennsylvania has announced an expanded relationship with Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating, genetic diseases.

21 Jan 2015

Researchers reveal key factor in understanding elevated cancer risk linked to gene therapy

National Institutes of Health researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome Research Institute, part of NIH, published their research in the Jan. 20, 2015, online issue of the Journal of Clinical Investigation.

21 Jan 2015

Researchers uncover new gene tied to incurable eye disorder

An exhaustive hereditary analysis of a large Louisiana family with vision issues has uncovered a new gene tied to an incurable eye disorder called retinitis pigmentosa, according to an examination led by scientists at The University of Texas Health Science Center at Houston.

16 Jan 2015

Scientists find that blocking hormone reduces stress-induced infertility in female rats

University of California, Berkeley, scientists have discovered that chronic stress activates a hormone that reduces fertility long after the stress has ended, and that blocking this hormone returns female reproductive behavior to normal.

13 Jan 2015

New UCLA study sheds light on why some people develop PTSD

Why do some people develop post-traumatic stress disorder (PTSD) while others who suffered the same ordeal do not? A new UCLA discovery may shed light on the answer.

11 Jan 2015

New findings may lay groundwork of novel treatment for people with Huntington's disease

By adjusting the levels of a key signaling protein, researchers improved motor function and brain abnormalities in experimental animals with a form of Huntington's disease, a severe neurodegenerative disorder.

5 Jan 2015

Spark Therapeutics files Form S-1 registration statement for initial public offering of common stock

Spark Therapeutics announced today that it has filed a registration statement on Form S-1 with the U.S. Securities and Exchange Commission relating to the proposed initial public offering of its common stock.

2 Jan 2015

New therapeutic approach shows potential for treating cancer

Case Western Reserve researchers have identified a two-pronged therapeutic approach that shows great potential for weakening and then defeating cancer cells.

31 Dec 2014

Researchers treat myocardial infarction with new telomerase-based gene therapy

The enzyme telomerase repairs cell damage produced by ageing, and has been used successfully in therapies to lengthen the life of mice. Now it has been observed that it could also be used to cure illnesses related to the ageing process.

18 Dec 2014

High-dose flu vaccine better than regular flu shot for frail, older adults of long-term care facilities

The high-dose flu vaccine is significantly better than the regular flu shot at boosting the immune response to the flu virus in frail, older residents of long-term care facilities, according to the results of a University of Pittsburgh School of Medicine study.

18 Dec 2014

Scientists demonstrate technique for editing genome in sperm-producing adult stem cells

Scientists at Indiana University and colleagues at Stanford and the University of Texas have demonstrated a technique for "editing" the genome in sperm-producing adult stem cells, a result with powerful potential for basic research and for gene therapy.

16 Dec 2014

Study finds that K13 gene mutations cause malaria drug resistance in Southeast Asia

Growing resistance to malaria drugs in Southeast Asia is caused by a single mutated gene inside the disease-causing Plasmodium falciparum parasite, according to a study led by David Fidock, PhD, professor of microbiology & immunology and of medical sciences (in medicine) at Columbia University Medical Center.

12 Dec 2014