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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.
Neurosurgeons develop new technologies to perform highly complex brain surgery

Neurosurgeons develop new technologies to perform highly complex brain surgery

In a milestone procedure, neurosurgeons at UC San Diego Health System have integrated advanced 3D imaging, computer simulation and next-generation surgical tools to perform a highly complex brain surgery through a small incision to remove deep-seated tumors. [More]
WIRB-Copernicus Group acquires Alliance Biosciences

WIRB-Copernicus Group acquires Alliance Biosciences

WIRB-Copernicus Group, the world's largest provider of regulatory and ethical review services for clinical research, announced today that it has acquired Alliance Biosciences. Formerly a division of Richmond, VA-based Alliance Engineering, Alliance Biosciences was the leading biosafety and biosecurity consulting firm in the United States. [More]
Parkinson's patients that respond favorably to sham surgery express distinct neurological profile

Parkinson's patients that respond favorably to sham surgery express distinct neurological profile

Parkinson's disease is the second most common neurological disorder, affecting almost 10 million people worldwide. [More]
Brain network measures placebo effects in Parkinson's disease patients

Brain network measures placebo effects in Parkinson's disease patients

Investigators at The Feinstein Institute for Medical Research have utilized a new image-based strategy to identify and measure placebo effects in randomized clinical trials for brain disorders. The findings are published in the August issue of The Journal of Clinical Investigation. [More]
Researchers find viable immunotherapy option for HIV-1 using fossil virus

Researchers find viable immunotherapy option for HIV-1 using fossil virus

The road to finding a cure for HIV-1 is not without obstacles. However, thanks to cutting-edge research by Douglas Nixon, M.D., Ph.D., and colleagues, performed at the George Washington University (GW), Oregon Health & Science University, the University of Rochester, and UC San Francisco, the scientific community is one step closer to finding a viable immunotherapy option for HIV-1, using an immune attack against a fossil virus buried in the genome. [More]
Splice-switching oligonucleotide drugs alter editing of gene transcript

Splice-switching oligonucleotide drugs alter editing of gene transcript

In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. [More]
Researchers devise new method for extracting molecules from live cells

Researchers devise new method for extracting molecules from live cells

University of Houston researchers have devised a new method for extracting molecules from live cells without disrupting cell development, work that could provide new avenues for the diagnosis of cancer and other diseases. [More]
Gene therapy offers potential alternative to pacemakers

Gene therapy offers potential alternative to pacemakers

Researchers have developed a gene transplant procedure that can transform ordinary heart muscle cells into “biological pacemaker” cells that maintain heart rhythm. [More]
New approach for developing personalized gene therapies to treat retinitis pigmentosa

New approach for developing personalized gene therapies to treat retinitis pigmentosa

Columbia University Medical Center researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss. [More]
Gene therapy can improve regenerative capacity of injured RGCs

Gene therapy can improve regenerative capacity of injured RGCs

Because the adult mammalian central nervous system has only limited intrinsic capacity to regenerate connections after injury, due to factors both intrinsic and extrinsic to the mature neuron, therapies are required to support the survival of injured neurons and to promote the long-distance regrowth of axons back to their original target structures. [More]
New gene therapy may be effective for fighting fungal infections in cancer patients

New gene therapy may be effective for fighting fungal infections in cancer patients

Sleeping Beauty and fungal infections - not two items one would normally associate together, but for immunocompromised cancer patients they may prove to be a helpful combination. [More]
Penn’s immunotherapy receives FDA's Breakthrough Therapy designation for ALL treatment

Penn’s immunotherapy receives FDA's Breakthrough Therapy designation for ALL treatment

A University of Pennsylvania-developed personalized immunotherapy has been awarded the U.S. Food and Drug Administration's Breakthrough Therapy designation for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL). [More]
Findings provide new insights into the basic biology of stem cells

Findings provide new insights into the basic biology of stem cells

A team of researchers from the University of California, San Diego School of Medicine, Oregon Health & Science University and Salk Institute for Biological Studies has shown for the first time that stem cells created using different methods produce differing cells. [More]
New gene therapy for Acute Intermittent Porphyria

New gene therapy for Acute Intermittent Porphyria

Angeles suffers from a severe and rare genetic disease called Acute Intermittent Porphyria (AIP). This means, one of her genes restrains her liver to produce a specific protein needed for the metabolism of the blood. During an AIP attack Angeles usually feels intense abdominal pain, and her mental status changes. Women live at an increased risk to trigger the symptoms, because porphyria is related to the menstrual cycle. [More]
Complete analysis of regenerative medicine market

Complete analysis of regenerative medicine market

According to a new market research report by Allied Market Research titled, "GlobalRegenerative MedicineMarket (Technology, Applications, Geography) - Industry Analysis, Trends, Opportunities and Forecast, 2013-2020", the global regenerative medicine market will reach $ 67.6 billion by 2020 from $16.4 billion in 2013, registering a CAGR of 23.2% during forecast period (2014 - 2020). [More]
Cancer drug shows promise in treating blood cell disorders

Cancer drug shows promise in treating blood cell disorders

Scientists working to make gene therapy a reality have solved a major hurdle: how to bypass a blood stem cell's natural defenses and efficiently insert disease-fighting genes into the cell's genome. [More]
Frederic Bushman gets Pioneer Award from Human Gene Therapy

Frederic Bushman gets Pioneer Award from Human Gene Therapy

Frederic D. Bushman, PhD's (University of Pennsylvania Perelman School of Medicine) early pioneering work in understanding how HIV reproduces by inserting its genetic material into the DNA of a host cell led to key advances in the ability to move pieces of DNA and whole genes between cells. [More]
New method to help patients with cancer, genetic conditions, neurological disorders

New method to help patients with cancer, genetic conditions, neurological disorders

UT Arlington physics researchers may have developed a way to use laser technology to deliver drug and gene therapy at the cellular level without damaging surrounding tissue. The method eventually could help patients suffering from genetic conditions, cancers and neurological diseases. [More]
Cardium reports positive results from Generx Ad5FGF-4 Phase 3 study for treatment of myocardial ischemia

Cardium reports positive results from Generx Ad5FGF-4 Phase 3 study for treatment of myocardial ischemia

Taxus Cardium Pharmaceuticals Group Inc. will report today at the Biotechnology Industry Organization’s 2014 BIO International Convention on a pre-specified interim analysis from its international ASPIRE Phase 3 registration clinical study of Generx Ad5FGF-4, Cardium Therapeutics’ angiogenic gene therapy product candidate for patients with myocardial ischemia due to coronary artery disease. [More]
Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Bayer, Dimension Therapeutics partner to develop new gene therapy for hemophilia A

Dimension Therapeutics, a company focused on developing novel adeno-associated virus (AAV) gene therapy treatments for rare diseases, today announced it has entered into a collaboration with Bayer HealthCare (Bayer) for the development and commercialization of a novel gene therapy for the treatment of hemophilia A. [More]