Gene Transfer News and Research

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

Researchers discover evolutionary nature of bacteria to spread chronic infectious disease

Researchers from the Center for Genomic Sciences at Allegheny General Hospital (AGH) in Pittsburgh have made a landmark discovery about the evolutionary nature of bacteria in the setting of chronic infectious disease. Reporting today in the journal PLos Pathogens, the AGH team documents for the first time that bacteria engage in a process called horizontal gene transfer to evolve rapidly during the course of a single infection.

18 Sep 2010

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

16 Sep 2010

Scientists reveal techniques to extend organ transplant life

Experts from the Medical Research Council (MRC) Centre for Transplantation at King's College London, based at Guy's Hospital, have revealed exciting new scientific developments for people with an organ transplant, intended to help prevent rejection of the new organ and extend its life.

16 Sep 2010

Scientists develop direct method to detect gene doping

Scientists at the universities in Tübingen and Mainz have developed a test that can provide conclusive proof of gene doping. "For the first time, a direct method is now available that uses conventional blood samples to detect doping via gene transfer and is still effective if the actual doping took place up to 56 days before," Professor Perikles Simon, MD, PhD from Johannes Gutenberg University Mainz, Germany explained on Thursday.

3 Sep 2010

Scientists replace human gene to preserve photoreceptor function in retinal degeneration

The easy accessibility of the eye and the established link between specific genetic defects and ocular disorders offer hope for using gene therapy to provide long-term therapeutic benefit. Two reports in the current issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc., describe the effective replacement of a human gene to preserve photoreceptor function in a mouse model of severe retinal degeneration. The articles are available free online.

16 Aug 2010

Neurologix second-quarter net loss increases to $4.5 million

Neurologix, Inc., a biotechnology company developing innovative gene therapies for the brain and central nervous system, today reported its financial results for the three and six months ended June 30, 2010.

13 Aug 2010

SORT1 pathway holds promise as new target for therapeutic intervention for LDL cholesterol reduction

The true power of genomic research lies in its ability to help scientists understand biological processes, particularly those that - when altered - can lead to disease. This power is demonstrated dramatically in a pair of papers published today in the journal Nature. In the first, a global team of researchers describes 95 different variations across the genome that contribute in different degrees to alterations in blood cholesterol and triglyceride levels in multiple human populations. In the second report, close examination of just one of these common variants not only reveals the involvement of an unexpected genetic pathway in lipid metabolism but also provides a blueprint for using genomic findings to unravel biological connections between lipid levels and coronary heart disease.

5 Aug 2010

Improved delivery methods needed to advance gene therapy strategies for prostate cancer: Review

Improved delivery methods and better testing systems are needed to advance promising gene therapy strategies for treating prostate cancer, according to a series of review articles in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc.

14 Jul 2010

Gene therapy represents safe alternative to current cures for blood disorder β-thalassemia

Italian scientists pioneering a new gene transfer treatment for the blood disorder β-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin (β-globin) in patients' blood cells which causes the disease. The research, published in EMBO Molecular Medicine, reveals how gene therapy may represent a safe alternative to current cures that are limited to a minority of patients.

14 Jul 2010

Scientists shrink distant sarcomas with genetically reprogrammed herpes virus and anti-vascular drug

Scientists have used a genetically reprogrammed herpes virus and an anti-vascular drug to shrink spreading distant sarcomas designed to model metastatic disease in mice – still an elusive goal when treating humans with cancer, according to a study in the July 8 Gene Therapy.

8 Jul 2010

Scientists use genetically reprogrammed herpes virus to shrink distant sarcomas

Scientists have used a genetically reprogrammed herpes virus and an anti-vascular drug to shrink spreading distant sarcomas designed to model metastatic disease in mice - still an elusive goal when treating humans with cancer, according to a study in the July 8 Gene Therapy.

8 Jul 2010

Gene therapy holds promise in treating cancer, cardiovascular and neurodegenerative diseases

EUREKA project E! 3371 Gene Transfer Agents has made great advances in the development of novel non-viral carriers able to introduce genetic material into the target cells.

25 Jun 2010

Neurologix announces positive results from Phase 2 trial of NLX-P101 for advanced PD

Neurologix, Inc., today announced positive results in a Phase 2 trial of its investigational gene therapy for advanced Parkinson's disease, NLX-P101. Study participants who received NLX-P101 experienced statistically significant and clinically meaningful improvements in off-medication motor scores compared to control subjects who received sham surgery.

22 Jun 2010

Novel gene replacement therapy holds promise for Spinal Muscular Atrophy

Children who suffer from the devastating disease Spinal Muscular Atrophy are set to benefit from a new breakthrough in therapy developments by researchers at the University of Sheffield.

10 Jun 2010

NIH, FDA launch new portal to provide easier access to online reporting

The Food and Drug Administration and the National Institutes of Health today launched a new Web site that, when fully developed, will provide a mechanism for the reporting of pre- and post-market safety data to the federal government.

25 May 2010

Lentigen receives NIH STTR grant for Hunter syndrome lentiviral gene therapy

Lentigen Corporation, a biotechnology company specializing in the development and manufacture of lentiviral gene delivery technologies, announced today that it has received a National Institutes of Health (NIH) small business technology transfer (STTR) grant for a program on "Lentiviral Gene Therapy for Mucopolysaccharidosis."

19 May 2010

Neurologix reports lower net loss of $3.5M for first-quarter 2010

Neurologix, Inc., a biotechnology company developing innovative gene therapies for the brain and central nervous system, today announced its financial results for the quarter ended March 31, 2010.

14 May 2010

Pain Therapeutics reports net loss of $1.0 million for first-quarter 2010

Pain Therapeutics, Inc., a biopharmaceutical company, today reported first quarter 2010 financial results. Net loss for Q1 2010 was $1.0 million, or $0.02 per share, compared to a net loss of $1.8 million, or $0.04 per share, for Q1 2009.

29 Apr 2010

New gene therapy to restore retinal cone function and day vision

Veterinary ophthalmology researchers from the University of Pennsylvania have used gene therapy to restore retinal cone function and day vision in two canine models of congenital achromatopsia, also called rod monochromacy or total color blindness.

22 Apr 2010

Researchers commence Phase 2 research study to test CERE-110 for AD

Researchers at Banner Sun Health Research Institute are about to start the first Phase 2 research study to test a gene transfer agent for Alzheimer's disease. AD is a degenerative and ultimately fatal disorder affecting as many as five million Americans and that number is expected to soar to more than 11 million by 2040.

7 Apr 2010