Gene Transfer News and Research

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Gene Transfer is the insertion of genetic material into a cell.

UIC researchers discover molecular switch that allows salmonella bacteria to fight immune system

Researchers at the University of Illinois at Chicago have discovered a molecular regulator that allows salmonella bacteria to switch from actively causing disease to lurking in a chronic but asymptomatic state called a biofilm.

7 Feb 2016

Leading researchers reject proposed link between adeno-associated virus 2 and hepatocellular carcinoma

The conclusion drawn from a recent study that insertion of adeno-associated virus 2 (AAV2) into human DNA causes mutations leading to the development of hepatocellular carcinoma (HCC) was resoundingly rejected by leading researchers in the fields of gene therapy and molecular genetics.

7 Jan 2016

Oxford University researchers unlock the secret behind bacterial sex

Migration between different communities of bacteria is the key to the type of gene transfer that can lead to the spread of traits such as antibiotic resistance, according to researchers at Oxford University.

23 Nov 2015

CHOP researchers delay symptoms, extend lifespan in animal model of Batten disease

Researchers have taken a significant step forward in developing gene therapy against a fatal neurodegenerative disease that strikes children. By delivering a working version of a gene to produce a key enzyme that is lacking in Batten disease, the scientists delayed symptoms and extended lifespan in dogs with a comparable disease.

12 Nov 2015

New gene therapy approach to treating DMD reduces symptoms, extends life span in mouse model

A gene therapy approach to treating the progressive muscle wasting disorder Duchenne muscular dystrophy (DMD) that does not replace the mutated DMD gene but instead delivers the gene for ITGA7, a protein in skeletal muscle, led to reduced symptoms and significantly extended life span in a mouse model of severe DMD. Over-expression of ITGA7 did not elicit an immune reaction, further supporting its potential as a novel treatment for DMD, according to a new study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

30 Sep 2015

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

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.

1 Sep 2015

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.

1 Sep 2015

Rice University scientists identify genetic mechanism that allows bacteria to resist antibiotics

Rice University scientists are developing strategies to keep germs from evolving resistance to antibiotics by heading them off at the pass.

5 Aug 2015

First biodegradable gene delivery system efficiently penetrates human airway mucus barrier of lung tissue

Researchers at the Johns Hopkins University School of Medicine, Johns Hopkins University Department of Chemical and Biomolecular Engineering, and Federal University of Rio de Janeiro in Brazil have designed a DNA-loaded nanoparticle that can pass through the mucus barrier covering conducting airways of lung tissue — proving the concept, they say, that therapeutic genes may one day be delivered directly to the lungs to the levels sufficient to treat cystic fibrosis (CF), chronic obstructive pulmonary disease, asthma and other life-threatening lung diseases.

4 Aug 2015

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.

31 Jul 2015

Acceleration of cell cycle transition kinetic can make human blood stem cells more powerful

For the first time, the research group of Prof. Claudia Waskow at the Carl Gustav Carus Faculty of Medicine at Dresden Technical University is now describing a new mechanism in which the length of the G1 phase of the cell cycle has a dramatic impact on the fitness of human blood stem cells.

13 Jul 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

University of Bonn researchers find way to stimulate larynx muscles using light

Researchers at the University of Bonn have found a way to stimulate the larynx muscles of mice using light. In the long term, this method could be an option for the treatment of laryngeal paralysis, which causes difficulties in phonation and breathing. Their findings will be published in the scientific journal "Nature Communications."

3 Jun 2015

FDA advisory committees to review Amgen's talimogene laherparepvec for metastatic melanoma treatment

Amgen announced today that the Cellular, Tissue and Gene Therapies Advisory Committee and the Oncologic Drugs Advisory Committee of the U.S. Food and Drug Administration will jointly review the Company's Biologics License Application (BLA) for talimogene laherparepvec.

11 Feb 2015

Two researchers receive Pioneer Award from Human Gene Therapy

Recognized for their pioneering work in the development of gene transfer technology using retroviral vectors to deliver therapeutic genes into cells, Richard C. Mulligan, PhD, Director of the Harvard Gene Therapy Initiative, Harvard Institutes of Medicine, Boston, MA, and A. Dusty Miller, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA, received the Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

9 Feb 2015

Intrexon and ZIOPHARM Oncology sign exclusive licensing agreement with MD Anderson

Intrexon Corporation, a leader in synthetic biology and its oncology partner, ZIOPHARM Oncology, today announced a broad exclusive licensing agreement with The University of Texas MD Anderson Cancer Center, including an exclusive sublicensing agreement through MD Anderson for intellectual property developed at the University of Minnesota for the development of non-viral adoptive cellular cancer immunotherapies.

14 Jan 2015

Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

Spark Therapeutics, a late-stage gene therapy company developing treatments for debilitating genetic diseases, announced today that it has entered into a global collaboration with Pfizer Inc. for the development and potential commercialization of SPK-FIX, a development program advancing proprietary, bio-engineered adeno-associated virus (AAV) vectors for the potential treatment of hemophilia B.

8 Dec 2014

Authors review current progress in developing transgenic pig models for human diseases

Genetically engineered pigs, minipigs, and microminipigs are valuable tools for biomedical research, as their lifespan, anatomy, physiology, genetic make-up, and disease mechanisms are more similar to humans than the rodent models typically used in drug discovery research.

24 Nov 2014

Study shows how stem cells can help regenerate damaged muscle after heart attack

Delivering stem cell factor directly into damaged heart muscle after a heart attack may help repair and regenerate injured tissue, according to a study led by researchers from Icahn School of Medicine at Mount Sinai presented November 18 at the American Heart Association Scientific Sessions 2014 in Chicago, IL.

19 Nov 2014

Gene therapy transforms life for men with severe form of hemophilia B

Gene therapy developed at St. Jude Children's Research Hospital, University College London and the Royal Free Hospital has transformed life for men with a severe form of hemophilia B by providing a safe, reliable source of the blood clotting protein Factor IX that has allowed some to adopt a more active lifestyle, researchers reported.