Gene Transfer News and Research

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

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.

19 Nov 2014

Researchers discover cardiac molecule that could provide key to treating, preventing heart failure

Researchers have discovered a previously unknown cardiac molecule that could provide a key to treating, and preventing, heart failure.

11 Aug 2014

WIRB-Copernicus Group acquires Alliance Biosciences

WIRB-Copernicus Group, the world's largest provider of regulatory and ethical review services for clinical research, announced today that it has acquired Alliance Biosciences. Formerly a division of Richmond, VA-based Alliance Engineering, Alliance Biosciences was the leading biosafety and biosecurity consulting firm in the United States.

24 Jul 2014

Splice-switching oligonucleotide drugs alter editing of gene transcript

In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein.

18 Jul 2014

Gene therapy offers potential alternative to pacemakers

Researchers have developed a gene transplant procedure that can transform ordinary heart muscle cells into “biological pacemaker” cells that maintain heart rhythm.

17 Jul 2014

New gene therapy may be effective for fighting fungal infections in cancer patients

Sleeping Beauty and fungal infections - not two items one would normally associate together, but for immunocompromised cancer patients they may prove to be a helpful combination.

9 Jul 2014

Frederic Bushman gets Pioneer Award from Human Gene Therapy

Frederic D. Bushman, PhD's (University of Pennsylvania Perelman School of Medicine) early pioneering work in understanding how HIV reproduces by inserting its genetic material into the DNA of a host cell led to key advances in the ability to move pieces of DNA and whole genes between cells.

25 Jun 2014

Researchers solve structure of key protein that drives DNA copying in plasmids

Staph infections that become resistant to multiple antibiotics don't happen because the bacteria themselves adapt to the drugs, but because of a kind of genetic parasite they carry called a plasmid that helps its host survive the antibiotics.

10 Jun 2014

Leukemia researcher wins 2014 Taubman Prize for Excellence in Translational Medical Science

A physician-scientist who developed a personalized immunotherapy for leukemia using patients' own T cells is the recipient of the 2014 Taubman Prize for Excellence in Translational Medical Science, awarded by the A. Alfred Taubman Medical Research Institute at the University of Michigan Medical School.

4 Jun 2014

Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords of animal models of SMA.

23 May 2014