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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.
USF, FARA to jointly host scientific symposium on Friedreich's ataxia

USF, FARA to jointly host scientific symposium on Friedreich's ataxia

The University of South Florida will again bring together leading researchers and patients searching for a treatment for Friedreich's ataxia and related disorders at the seventh annual scientific symposium "Understanding Energy for A Cure." The symposium will be held 5 to 8:30 p.m., Thursday, Sept. 17, at the USF Marshall Student Center Ballroom, USF Cedar Circle, Tampa, FL 33620. [More]
Affinity tuning can make CAR T cells spare normal cells and attack cancer cells

Affinity tuning can make CAR T cells spare normal cells and attack cancer cells

A new development in engineering chimeric antigen receptor (CAR) T cells, called affinity tuning, can make the CAR T cells spare normal cells and better recognize and attack cancer cells, which may help lower the toxicity associated with this type of immunotherapy when used against solid tumors, according to a preclinical study. [More]
Gene transfer therapy not beneficial for heart failure patients with reduced ejection fraction

Gene transfer therapy not beneficial for heart failure patients with reduced ejection fraction

Gene transfer therapy aimed at correcting an enzyme abnormality involved in myocardial contraction and relaxation did not improve outcomes in heart failure patients with reduced ejection fraction, results of the CUPID 2 study show. [More]
Gene therapy restores visual function in mouse model of LCA1

Gene therapy restores visual function in mouse model of LCA1

Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. [More]
CUMC vision researchers discover gene that causes myopia

CUMC vision researchers discover gene that causes myopia

Vision researchers at Columbia University Medical Center have discovered a gene that causes myopia, but only in people who spend a lot of time in childhood reading or doing other "nearwork." [More]
Researchers identify new virus that plays role in rare type of liver cancer

Researchers identify new virus that plays role in rare type of liver cancer

More than a cause of a simple infection, viruses are often involved in the development of serious diseases. Such is the case with liver cancer, which often develops in an organ that has been weakened by hepatitis B or C virus. [More]
Experimental gene therapy may prevent neuronal degeneration in patients with Alzheimer's disease

Experimental gene therapy may prevent neuronal degeneration in patients with Alzheimer's disease

Degenerating neurons in patients with Alzheimer's disease (AD) measurably responded to an experimental gene therapy in which nerve growth factor (NGF) was injected into their brains, report researchers at University of California, San Diego School of Medicine in the current issue of JAMA Neurology. [More]
Gene delivery services expanded for adeno-associated virus

Gene delivery services expanded for adeno-associated virus

AMSBIO has introduced an expanded range of Adeno-associated virus (AAV) cloning and packaging services. [More]
Benitec Biopharma announces closing of U.S. initial public offering of ADSs

Benitec Biopharma announces closing of U.S. initial public offering of ADSs

Benitec Biopharma Limited, a clinical-stage biotechnology company, is pleased to announce the closing of its U.S. initial public offering of 1,500,000 American Depositary Shares (ADSs), representing 30,000,000 fully paid ordinary shares of Benitec, together with warrants to purchase 500,000 ADSs, representing 10,000,000 fully paid ordinary shares. [More]
Stanford University launches new professional education course

Stanford University launches new professional education course

Imagine a world where health care is personalized, diseases are treated or even prevented before they become debilitating, and doctor's visits include regular reviews of individual genetic profiles. These are just some of the possibilities at the intersection of healthcare and genomics. [More]
MCRI announces winners of cancer crowdsourcing initiative

MCRI announces winners of cancer crowdsourcing initiative

The Myeloma Crowd Research Initiative (MCRI), an unprecedented collaboration of cancer researchers and patient advocates, today announced the two winners of its first-ever crowdsourcing and patient-led initiative to fund research in high-risk multiple myeloma, a rare malignancy of plasma cells. [More]
Benitec prices initial public offering of 1,500,000 American Depositary Shares

Benitec prices initial public offering of 1,500,000 American Depositary Shares

Benitec Biopharma Limited, a clinical-stage biotechnology company, is pleased to announce the pricing of its U.S. initial public offering of 1,500,000 American Depositary Shares (ADSs), representing 30,000,000 fully paid ordinary shares of Benitec and warrants to purchase 500,000 ADSs, representing 10,000,000 fully paid ordinary shares, at a price of US$9.21 per ADS and US$0.01 per warrant. [More]
Researchers use advanced photodynamic therapy to combat ovarian cancer in laboratory animals

Researchers use advanced photodynamic therapy to combat ovarian cancer in laboratory animals

Researchers at Oregon State University have made a significant advance in the use of photodynamic therapy to combat ovarian cancer in laboratory animals, using a combination of techniques that achieved complete cancer cell elimination with no regrowth of tumors. [More]
Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale inspires new law to create treatments for rare diseases, spur growth in NC

Charlotte charity Taylor's Tale helped inspire a law designed to stimulate the creation of new treatments for rare diseases and spur economic development in North Carolina. [More]
NFIX protein drives NSC differentiation toward oligodendrocytes

NFIX protein drives NSC differentiation toward oligodendrocytes

An international team of researchers has shown that NFIX, a protein that regulates neuronal stem cell activity (NSC), also has a role in driving NSC differentiation toward oligodendrocytes, a type of glial cell. These cells produce the myelin that surrounds and protects neurons. [More]
University of Zurich professor recognized with 2015 Vallee Young Investigator Award

University of Zurich professor recognized with 2015 Vallee Young Investigator Award

Martin Jinek, a professor at the University of Zurich's Department of Biochemistry, was presented with the 2015 Vallee Young Investigator Award. The international prize is awarded to young researchers for outstanding achievements in biomedicine and carries USD 250,000 in prize money. [More]
Penn Medicine devises new approach to develop vaccines against lethal diseases

Penn Medicine devises new approach to develop vaccines against lethal diseases

Researchers at the Perelman School of Medicine at the University of Pennsylvania have devised an entirely new approach to vaccines - creating immunity without vaccination. [More]
Researchers reconstruct ancient virus to improve gene therapy

Researchers reconstruct ancient virus to improve gene therapy

Researchers at Massachusetts Eye and Ear/Schepens Eye Research Institute have reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle, and retina. This discovery, published July 30 in Cell Reports, could potentially be used to design gene therapies that are not only safer and more potent than therapies currently available, but may also help a greater number of patients. [More]
Novel 3D human skin tissue model could help detect presence of known skin sensitizers in medical device extracts

Novel 3D human skin tissue model could help detect presence of known skin sensitizers in medical device extracts

New research shows that exposing a 3D human skin tissue model to extracts of medical device materials can detect the presence of sensitizers known to cause an allergic response on contact in some individuals. Conventional skin sensitization testing of medical devices relies on animal testing, whereas human skin models could replace animal methods, according to an article in the new journal Applied In Vitro Toxicology, a peer-reviewed publication from Mary Ann Liebert, Inc., publishers. [More]
Innovative approach to treating AAT deficiency

Innovative approach to treating AAT deficiency

Researchers have demonstrated the feasibility of delivering an RNA that encodes for the protein alpha-1-antitrypsin (AAT)--which is missing or nonfunctional in the genetic disorder AAT deficiency--into cells in the laboratory, enabling the cells to produce highly functional AAT. [More]
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