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.

Researchers identify previously unknown gene mutation that causes achromatopsia

People with achromatopsia, an inherited eye disorder, see the world literally in black and white. Worse yet, their extreme sensitivity to light makes them nearly blind in bright sunlight. Now, researchers at University of California, San Diego School of Medicine and Shiley Eye Institute at UC San Diego Health System have identified a previously unknown gene mutation that underlies this disorder.

2 Jun 2015

Adaptimmune presents clinical results of NY-ESO TCR therapeutic candidate at ASCO 2015

Adaptimmune Therapeutics plc, a clinical stage biopharmaceutical company focused on the use of TCR engineered T-cell therapy to treat cancer, today announced a poster presentation of data on its lead clinical program, an affinity enhanced T-cell receptor (TCR) therapeutic targeting the NY-ESO-1 cancer antigen, in both solid and hematologic cancers.

2 Jun 2015

Previously unknown gene mutation linked to inherited eye disorder, achromatopsia

2 Jun 2015

Six research teams awarded grant to accelerate discovery of new drugs for brain, nervous system disorders

CQDM, Brain Canada and the Ontario Brain Institute award close to $8.5M to six (6) multi-disciplinary and multi-provincial research teams across Canada to address unmet needs in neuroscience within their Focus on Brain strategic initiative. To this amount, $1.5M is added from the various research entities involved as in-kind contributions.

27 May 2015

New stem-cell based therapy provides pain relief, reduces severity of RDEB in children

Promising results from a trial of a new stem-cell based therapy for a rare and debilitating skin condition have been published in the Journal of Investigative Dermatology. The therapy, involving infusions of stem cells, was found to provide pain relief and to reduce the severity of this skin condition for which no cure currently exists.

27 May 2015

UVA scientists find blueprint for combating human disease using DNA clad in near-indestructible armor

By unlocking the secrets of a bizarre virus that survives in nearly boiling acid, scientists at the University of Virginia School of Medicine have found a blueprint for battling human disease using DNA clad in near-indestructible armor.

27 May 2015

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.

15 May 2015

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.

14 May 2015

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.

14 May 2015

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.

12 May 2015

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.

11 May 2015

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.

8 May 2015

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.

30 Apr 2015

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.

30 Apr 2015

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

29 Apr 2015

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.

29 Apr 2015

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.

28 Apr 2015

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.

22 Apr 2015

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.

21 Apr 2015

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.

21 Apr 2015

Gene Therapy News and Research

Further Reading

Researchers identify previously unknown gene mutation that causes achromatopsia

Adaptimmune presents clinical results of NY-ESO TCR therapeutic candidate at ASCO 2015

Previously unknown gene mutation linked to inherited eye disorder, achromatopsia

Six research teams awarded grant to accelerate discovery of new drugs for brain, nervous system disorders

New stem-cell based therapy provides pain relief, reduces severity of RDEB in children

UVA scientists find blueprint for combating human disease using DNA clad in near-indestructible armor

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

Small-molecule drug simultaneously rejuvenates old muscles, aging brains

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

Experimental gene therapy holds promise against metastatic prostate cancer

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

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

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

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

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

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

Yale researchers successfully correct gene mutation that causes cystic fibrosis

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

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

Global changes in cancer cells' epigenome may determine disease progression

Leveraging the power of automation to boost research

Automating research - solutions and best practices

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