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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.
Chemical drug safely controls side effects associated with haploidentical stem cell transplantation

Chemical drug safely controls side effects associated with haploidentical stem cell transplantation

Researchers in the Center for Cell and Gene Therapy at Baylor College of Medicine, Houston Methodist and Texas Children's Hospital have found that a single dose of an otherwise harmless drug can safely control the severe and often lethal side effects associated with haploidentical stem cell transplantation. [More]
Small-molecule drug simultaneously rejuvenates old muscles, aging brains

Small-molecule drug simultaneously rejuvenates old muscles, aging brains

Whether you're brainy, brawny or both, you may someday benefit from a drug found to rejuvenate aging brain and muscle tissue. Researchers at the University of California, Berkeley, have discovered that a small-molecule drug simultaneously perks up old stem cells in the brains and muscles of mice, a finding that could lead to drug interventions for humans that would make aging tissues throughout the body act young again. [More]
Non-coding RNAs in maternal food can pass through placenta to regulate fetal gene expression

Non-coding RNAs in maternal food can pass through placenta to regulate fetal gene expression

In a new study published in the Protein & Cell, Chen-Yu Zhang's group at Nanjing University reports that small non-coding RNAs in maternal food can transfer through placenta to regulate fetal gene expression. [More]
Experimental gene therapy holds promise against metastatic prostate cancer

Experimental gene therapy holds promise against metastatic prostate cancer

Even with the best available treatments, the median survival of patients with metastatic, hormone-refractory prostate cancer is only two to three years. Driven by the need for more effective therapies for these patients, researchers at VCU Massey Cancer Center and the VCU Institute of Molecular Medicine have developed a unique approach that uses microscopic gas bubbles to deliver directly to the cancer a viral gene therapy in combination with an experimental drug that targets a specific gene driving the cancer's growth. [More]
Dana-Farber/Boston Children's joins immunotherapy clinical trial for children with ALL

Dana-Farber/Boston Children's joins immunotherapy clinical trial for children with ALL

Dana-Farber/Boston Children's Cancer and Blood Disorders Center has joined a clinical trial of immunotherapy for children with relapsed or treatment-resistant acute lymphoblastic leukemia (ALL). Led by Memorial Sloan Kettering Cancer Center, the trial is one of several nationally that are evaluating cancer immunotherapy, a treatment approach -- hailed by Science magazine as their Breakthrough of the Year in 2013 -- that triggers a patient's immune system to attack his or her cancer cells. [More]
Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is the delivery of DNA into a patient's cells to replace faulty or missing genes—or adds new genes—in an attempt to cure cancer or make changes so the body is better able to fight off disease. Scientists and clinicians have identified a number of different ways to do this, in an effort to correct malfunctioning or mutated genes. Many gene therapy clinical trials are ongoing to assess the safety and potential benefits in patients with rare diseases. [More]
Study finds that gene therapy can clip out genetic material associated with heart failure

Study finds that gene therapy can clip out genetic material associated with heart failure

Gene therapy can clip out genetic material linked to heart failure and replace it with the normal gene in human cardiac cells, according to a study led by researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai. [More]
Study opens door for new therapeutic approaches to treating patients with melanoma

Study opens door for new therapeutic approaches to treating patients with melanoma

Weill Cornell Medical College researchers have shown for the first time that a gene previously implicated in blood vessel formation during embryonic development and tumor growth also induces immune suppression during tumor development. [More]
Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy [More]
Fifth patient dosed in Benitec's TT-034 Phase I/IIa clinical trial for prevention of HCV infection

Fifth patient dosed in Benitec's TT-034 Phase I/IIa clinical trial for prevention of HCV infection

Benitec Biopharma, a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or "expressed RNAi," is pleased to advise that the fifth patient in the company's 'first in man', Phase I/IIa dose escalation clinical trial of TT-034 for hepatitis C virus (HCV) infection, has today, been dosed at the Duke Clinical Research Unit. [More]
Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully correct gene mutation that causes cystic fibrosis

Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications. [More]
Gene therapy reduces infectious complications in children, teens with Wiskott-Aldrich syndrome

Gene therapy reduces infectious complications in children, teens with Wiskott-Aldrich syndrome

In a small study that included seven children and teens with Wiskott-Aldrich syndrome, a rare immunodeficiency disorder, use of gene therapy resulted in clinical improvement in infectious complications, severe eczema, and symptoms of autoimmunity, according to a study in the April 21 issue of JAMA, a theme issue on child health. [More]
UCLA-led stroke study selected as one of 10 most outstanding research papers by CRF

UCLA-led stroke study selected as one of 10 most outstanding research papers by CRF

A UCLA-led study on improving stroke care was selected by the Clinical Research Forum (CRF) as one of the 10 most outstanding research papers written by teams from across the nation in 2014. The organization highlighted the papers at its fourth annual awards ceremony in Washington, D.C. on April 16. [More]
Global changes in cancer cells' epigenome may determine disease progression

Global changes in cancer cells' epigenome may determine disease progression

Genomic studies have illuminated the ways in which malfunctioning genes can drive cancer growth while stunting the therapeutic effects of chemotherapy and other treatments. But new findings from Weill Cornell Medical College investigators indicate that these genes are only partly to blame for why treatment that was at one point effective ultimately fails for about 40 percent of patients diagnosed with the most common form of non-Hodgkin Lymphoma. [More]
New Dream Team dedicated to ovarian cancer research formed at AACR Annual Meeting 2015

New Dream Team dedicated to ovarian cancer research formed at AACR Annual Meeting 2015

Stand Up To Cancer, Ovarian Cancer Research Fund, Ovarian Cancer National Alliance, and National Ovarian Cancer Coalition, along with the American Association for Cancer Research, Scientific Partner to SU2C, announced today the formation of a Dream Team devoted to ovarian cancer research at the AACR Annual Meeting 2015, held here April 18-22. [More]
University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

University of Washington receives PPMD grant to continue analysis of spectrin-like repeats in dystrophin

Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne) awarded University of Washington a $148,000 grant to continue the functional analysis of spectrin-like repeats in dystrophin. [More]
RetroSense Therapeutics receives Luis Villalobos Award

RetroSense Therapeutics receives Luis Villalobos Award

RetroSense Therapeutics, LLC, a Wayne State University start-up biotechnology company, has received the Luis Villalobos Award from the Angel Capital Association, the world's leading professional association for angel investors. [More]
Donald S. Coffey to receive Margaret Foti Award at AACR Annual Meeting 2015

Donald S. Coffey to receive Margaret Foti Award at AACR Annual Meeting 2015

Donald S. Coffey, PhD, will be honored with the ninth annual American Association for Cancer Research Margaret Foti Award for Leadership and Extraordinary Achievements in Cancer Research at the AACR Annual Meeting 2015, to be held in Philadelphia, April 18-22. [More]
Autophagy: A new approach to fighting tuberculosis

Autophagy: A new approach to fighting tuberculosis

A new approach to combatting tuberculosis would take advantage of a complex, natural process called autophagy that the human body uses to recycle nutrients, remove damaged cell components, eliminate invading bacteria, and respond to inflammation. [More]
Penn researchers receive 2015 Vaccine Industry Excellence Award

Penn researchers receive 2015 Vaccine Industry Excellence Award

The laboratory of David Weiner, PhD, a professor of Pathology and Laboratory Medicine at the Perelman School of Medicine at the University of Pennsylvania, received the 2015 Vaccine Industry Excellence Award for Best Academic Research Team, at the World Vaccine Congress in Washington, DC this week. [More]
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