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Gene Transfer is the insertion of genetic material into a cell.
Spark Therapeutics, Pfizer partner to develop SPK-FIX for potential treatment of hemophilia B

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. [More]
Authors review current progress in developing transgenic pig models for human diseases

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. [More]
Study shows how stem cells can help regenerate damaged muscle after heart attack

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. [More]
Gene therapy transforms life for men with severe form of hemophilia B

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. [More]
Researchers discover cardiac molecule that could provide key to treating, preventing heart failure

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. [More]
WIRB-Copernicus Group acquires Alliance Biosciences

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. [More]
Splice-switching oligonucleotide drugs alter editing of gene transcript

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. [More]
Gene therapy offers potential alternative to pacemakers

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. [More]
New gene therapy may be effective for fighting fungal infections in cancer patients

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. [More]
Frederic Bushman gets Pioneer Award from Human Gene Therapy

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. [More]
Researchers solve structure of key protein that drives DNA copying in plasmids

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. [More]
Leukemia researcher wins 2014 Taubman Prize for Excellence in Translational Medical Science

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. [More]
Study: Sufficient copies of SMN1 gene extend survival in animals with spinal muscular atrophy

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. [More]
Manure from dairy cows contains newly identified antibiotic resistance genes

Manure from dairy cows contains newly identified antibiotic resistance genes

Manure from dairy cows, which is commonly used as a farm soil fertilizer, contains a surprising number of newly identified antibiotic resistance genes from the cows' gut bacteria. The findings, reported in mBio- the online open-access journal of the American Society for Microbiology, hints that cow manure is a potential source of new types of antibiotic resistance genes that transfer to bacteria in the soils where food is grown. [More]
Biologists find ways to address antibiotic resistance

Biologists find ways to address antibiotic resistance

Biologists could gain a deeper understanding about how species have evolved - and even find ways to address antibiotic resistance - using tools that were developed recently at Stockholm's KTH Royal Institute of Technology. [More]
Marina Cavazzana, Adrian J. Thrasher honored with Pioneer Award for gene therapy

Marina Cavazzana, Adrian J. Thrasher honored with Pioneer Award for gene therapy

Marina Cavazzana, MD, PhD, Paris Descartes University, France and Adrian J. Thrasher, MD, PhD, University College London Institute of Child Health, UK, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. [More]
Trimethoprim is more effective against streptococci than expected, says research

Trimethoprim is more effective against streptococci than expected, says research

The focus of his team was also on samples, in which the bacteria failed to respond to the agent. They discovered two types of resistance. "Spontaneous mutations can occur in the gene for dihydrofolate reductase rendering trimethoprim no longer able to attack the changed enzyme, which means it becomes ineffective," Nitsche-Schmitz explained. [More]
MD Anderson collaborates with MedImmune to develop therapies that unleash patients' immune systems to attack cancers

MD Anderson collaborates with MedImmune to develop therapies that unleash patients' immune systems to attack cancers

The University of Texas MD Anderson Cancer Center and MedImmune, the global biologics research and development arm of AstraZeneca, will collaborate through MD Anderson's Moon Shots Program to develop therapies that unleash patients' immune systems to attack their cancers. [More]
European Commission invests nearly $475 million in gene transfer and gene therapy projects

European Commission invests nearly $475 million in gene transfer and gene therapy projects

Over the past three funding stages, the European Commission has invested nearly $475 million in 100 projects in the gene transfer and gene therapy field. [More]
IRSF announces new research grants for translational research program and clinical trials

IRSF announces new research grants for translational research program and clinical trials

The International Rett Syndrome Foundation (IRSF) announces today ten (10) new research grants at leading global research institutions; a new Translational Research Program; and additional funding for current human clinical trials. [More]