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.

Newborn screening indicates that incidence of SCID is higher than previously believed

Newborn screening performed in numerous states indicates that the incidence of the potentially life-threatening disorder, severe combined immunodeficiency, is higher than previously believed, at 1 in 58,000 births, although there is a high rate of survival, according to a study in the August 20 issue of JAMA.

20 Aug 2014

Researchers use new gene editing method to correct mutation that leads to DMD

UT Southwestern Medical Center researchers successfully used a new gene editing method to correct the mutation that leads to Duchenne muscular dystrophy (DMD) in a mouse model of the condition.

17 Aug 2014

New x-ray imaging system helps monitor effectiveness of treatment for cystic fibrosis

Scientists have developed an x-ray imaging system that enables researchers to see 'live' how effective treatments are for cystic fibrosis.

16 Aug 2014

Oncothyreon acquires Alpine Biosciences to enable targeted delivery of multiple therapeutic agents

Oncothyreon Inc., a biotechnology company specializing in the development of innovative therapeutic products for the treatment of cancer, today announced that it has acquired Alpine Biosciences, Inc., of Seattle, Washington, a privately held biotechnology company developing protocells, a nanoparticle platform technology designed to enable the targeted delivery of multiple therapeutic agents, including nucleic acids, proteins, peptides and small molecules.

12 Aug 2014

Patient with mechanical heart pump receives new gene therapy for heart failure

For the first time in the world, a patient with a mechanical heart pump has received a new gene therapy for heart failure at Harefield Hospital, London.

11 Aug 2014

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists have discovered a new form of dystrophin, a protein critical to normal muscle function, and identified the genetic mechanism responsible for its production.

11 Aug 2014

Scientists find new clues to early detection, personalised treatment of ovarian cancer

Scientists at A*STAR's Institute of Medical Biology and the Bioinformatics Institute have found new clues to early detection and personalised treatment of ovarian cancer, currently one of the most difficult cancers to diagnose early due to the lack of symptoms that are unique to the illness.

9 Aug 2014

Vitro toxicity testing an alternative to predict adverse health effects of drugs

In vitro toxicity testing is rapidly being adopted in the pharmaceutical, chemical, and cosmetics industries, for example, as an alternative to animal studies to predict adverse health effects of drugs and personal care products and the health consequences of environmental exposures.

8 Aug 2014

Neurosurgeons develop new technologies to perform highly complex brain surgery

In a milestone procedure, neurosurgeons at UC San Diego Health System have integrated advanced 3D imaging, computer simulation and next-generation surgical tools to perform a highly complex brain surgery through a small incision to remove deep-seated tumors.

25 Jul 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

Parkinson's patients that respond favorably to sham surgery express distinct neurological profile

Parkinson's disease is the second most common neurological disorder, affecting almost 10 million people worldwide.

21 Jul 2014

Brain network measures placebo effects in Parkinson's disease patients

Investigators at The Feinstein Institute for Medical Research have utilized a new image-based strategy to identify and measure placebo effects in randomized clinical trials for brain disorders. The findings are published in the August issue of The Journal of Clinical Investigation.

19 Jul 2014

Researchers find viable immunotherapy option for HIV-1 using fossil virus

The road to finding a cure for HIV-1 is not without obstacles. However, thanks to cutting-edge research by Douglas Nixon, M.D., Ph.D., and colleagues, performed at the George Washington University (GW), Oregon Health & Science University, the University of Rochester, and UC San Francisco, the scientific community is one step closer to finding a viable immunotherapy option for HIV-1, using an immune attack against a fossil virus buried in the genome.

18 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

Researchers devise new method for extracting molecules from live cells

University of Houston researchers have devised a new method for extracting molecules from live cells without disrupting cell development, work that could provide new avenues for the diagnosis of cancer and other diseases.

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 approach for developing personalized gene therapies to treat retinitis pigmentosa

Columbia University Medical Center researchers have created a way to develop personalized gene therapies for patients with retinitis pigmentosa (RP), a leading cause of vision loss.

11 Jul 2014

Gene therapy can improve regenerative capacity of injured RGCs

Because the adult mammalian central nervous system has only limited intrinsic capacity to regenerate connections after injury, due to factors both intrinsic and extrinsic to the mature neuron, therapies are required to support the survival of injured neurons and to promote the long-distance regrowth of axons back to their original target structures.

10 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

Penn’s immunotherapy receives FDA's Breakthrough Therapy designation for ALL treatment

A University of Pennsylvania-developed personalized immunotherapy has been awarded the U.S. Food and Drug Administration's Breakthrough Therapy designation for the treatment of relapsed and refractory adult and pediatric acute lymphoblastic leukemia (ALL).

7 Jul 2014

Gene Therapy News and Research

Further Reading

Newborn screening indicates that incidence of SCID is higher than previously believed

Researchers use new gene editing method to correct mutation that leads to DMD

New x-ray imaging system helps monitor effectiveness of treatment for cystic fibrosis

Oncothyreon acquires Alpine Biosciences to enable targeted delivery of multiple therapeutic agents

Patient with mechanical heart pump receives new gene therapy for heart failure

Isoform: New protein offers novel therapeutic approach for patients with DMD

Scientists find new clues to early detection, personalised treatment of ovarian cancer

Vitro toxicity testing an alternative to predict adverse health effects of drugs

Neurosurgeons develop new technologies to perform highly complex brain surgery

WIRB-Copernicus Group acquires Alliance Biosciences

Parkinson's patients that respond favorably to sham surgery express distinct neurological profile

Brain network measures placebo effects in Parkinson's disease patients

Researchers find viable immunotherapy option for HIV-1 using fossil virus

Splice-switching oligonucleotide drugs alter editing of gene transcript

Researchers devise new method for extracting molecules from live cells

Gene therapy offers potential alternative to pacemakers

New approach for developing personalized gene therapies to treat retinitis pigmentosa

Gene therapy can improve regenerative capacity of injured RGCs

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

Penn’s immunotherapy receives FDA's Breakthrough Therapy designation for ALL treatment

Leveraging the power of automation to boost research

Automating research - solutions and best practices

How can microdialysis benefit drug development

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