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.

TSRI study reveals alternative approach to current anti-HIV strategies

HIV-infected patients remain on antiretroviral therapy for life because the virus survives over the long-term in infected dormant cells. Interruption of current types of antiretroviral therapy results in a rebound of the virus and clinical progression to AIDS.

9 Jul 2015

Findings reveal new pathway to develop effective treatments and therapies for asthma, allergy

Investigators have discovered the precise molecular steps that enable immune cells implicated in certain forms of asthma and allergy to develop and survive in the body. The findings from Weill Cornell Medical College reveal a new pathway that scientists could use to develop more effective treatments and therapies for the chronic lung disorder.

7 Jul 2015

Gene therapy improves lung function in patients with cystic fibrosis

Gene therapy was shown to provide significant benefits for patients with cystic fibrosis in a recent clinical study published in The Lancet Respiratory Medicine journal.

3 Jul 2015

Gene therapy for cystic fibrosis shows significant benefit in lung function

For the first time gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomised trial published in The Lancet Respiratory Medicine journal. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells.

3 Jul 2015

New NEI study shows that microglia can accelerate damage wrought by blinding eye disorders

Spider-like cells inside the brain, spinal cord and eye hunt for invaders, capturing and then devouring them. These cells, called microglia, often play a beneficial role by helping to clear trash and protect the central nervous system against infection. But a new study by researchers at the National Eye Institute (NEI) shows that they also accelerate damage wrought by blinding eye disorders, such as retinitis pigmentosa.

2 Jul 2015

Canada's first human gene therapy trial for choroideremia now underway at Royal Alexandra Hospital

Canada's first human gene therapy trial for eyes -- the replacement of a faulty gene with a healthy one -- is now underway at the Royal Alexandra Hospital to preserve and potentially restore vision for people with a genetic disorder that leaves them blind by middle age.

30 Jun 2015

Two physician-scientists in search for better Alzheimer's disease treatment

Two of the nation's leading physician-scientists in the search to better understand and treat Alzheimer's disease - William Mobley, MD, PhD, and Michael Rafii, MD, PhD - have been named interim co-directors of the Alzheimer's Disease Cooperative Study (ADCS), a major initiative formed in 1991 as a cooperative agreement between the National Institute on Aging (NIA) and the University of California, San Diego.

27 Jun 2015

Innovative gene transfer-based treatment for kids with giant axonal neuropathy

University of North Carolina School of Medicine researchers have developed an innovative, experimental gene transfer-based treatment for children with giant axonal neuropathy (GAN).

26 Jun 2015

Wistar Institute researchers identify specific mutations responsible for ineffectiveness of flu vaccines

Viruses like influenza have the ability to mutate over time, and given that the flu vaccines administered during the 2014-2015 season were largely ineffective at preventing the spread of the flu, it appears the virus that recently circulated had taken on mutations not accounted for when last year's vaccine was developed.

26 Jun 2015

WHO prequalifies new male circumcision device to prevent HIV

The ShangRing, a novel medical device for voluntary medical male circumcision, has received prequalification from the World Health Organization (WHO) for use.

25 Jun 2015

Israeli researchers establish novel optogenetic method for cardiac pacing and resynchronization

Israeli researchers have successfully established a new approach for pacing the heart and synchronizing its mechanical activity without the use of a conventional electrical pacemaker. This novel biologic strategy employs light-sensitive genes that can be injected into the heart and then activated by flashes of blue light.

23 Jun 2015

Benitec, ReNeuron collaborate to launch new exploratory cellular therapy program

Benitec Biopharma is pleased to announce the launch of a new exploratory cellular therapy program including exosome-based delivery utilising the Company's proprietary ddRNAi technology. Entry into these areas have been facilitated by the commencement of a collaboration with UK-based stem cell therapeutics company, ReNeuron.

22 Jun 2015

ExCellThera's stem cell expansion approach may benefit AML patients undergoing transplants

Patients suffering from acute myeloid leukemia, who require stem cell transplantation as part of their treatment,may now benefit from a new best-in-class process that improves the viability and success of cord blood stem cell transplantation.

18 Jun 2015

Researchers focus on novel techniques to combat global blood shortage

Red blood cells are the cells responsible for transporting oxygen to all the organs of the body. Red blood cells are the body's most common cell type: no less than 20 billion red blood cells die and are regenerated every day.

17 Jun 2015

FDA grants Fast Track designation to Juventas' JVS-100 drug candidate and allows Phase 2b clinical trial

Juventas Therapeutics, Inc., a clinical-stage biotechnology company developing novel non-viral gene therapies that activate natural processes to repair the body, today announced that its product candidate, JVS-100, received Fast Track designation from the U.S. Food and Drug Administration for the treatment of advanced ischemic chronic heart failure.

16 Jun 2015

Weill Cornell scientists reveal how XBP1 gene can trigger immune responses against ovarian tumors

Ovarian cancer shuts down immune system cells that would otherwise act as a first line of defense against the deadly tumor, Weill Cornell Medical College scientists report today. But a therapy that restores the cells' disease-fighting abilities could provide a powerful new strategy to attack the cancer, which kills more than 14,000 women each year.

12 Jun 2015

Creighton researcher receives NIH grant to study gene, stem cell therapy in coronary artery bypass grafts

A Creighton University researcher has received a National Institutes of Health grant to study the effects of gene and stem cell therapy in coronary artery bypass grafts, a first-of-its-kind undertaking that could transform the procedure, increase survival rates and dramatically reduce the possibility of re-occlusion of the grafted arteries and veins in the procedure.

10 Jun 2015

Novel strategy shows aptamer-based modular delivery of microRNA in endothelial, breast cancer cells

Researchers have shown that a novel delivery strategy can efficiently introduce a functional microRNA that has anti-cancer and angiogenic activities into two different types of cells--breast cancer cells to inhibit tumor growth and metastasis, and cells that line blood vessels to protect against atherosclerosis.

4 Jun 2015

BioLife reports increased use of CryoStor and HypoThermosol biopreservation media products in clinical studies

BioLife Solutions, Inc., a leading developer, manufacturer and marketer of proprietary clinical grade cell and tissue hypothermic storage and cryopreservation freeze media and a related cloud hosted biologistics cold chain management app for smart shippers, today reported several new customer disclosures on the use of the Company's CryoStor and HypoThermosol biopreservation media products in pre-clinical validation projects and clinical trials at the recent International Society for Cellular Therapy (ISCT) conference.

3 Jun 2015

Takara Bio Europe launches Cellartis DEF-CS Xeno-Free Culture Medium for iPS cell culture

Takara Bio Europe AB, formerly Cellartis AB, a wholly-owned subsidiary of Takara Bio Inc., announced today that it is launching an advanced cell culture medium for iPS cell culture. This chemically-defined culture medium is pre-clinical grade, free from human- and animal-derived components, and designed for efficient expansion of undifferentiated human iPS cells.