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.

New genetic cause of retinitis pigmentosa

Combining the expertise of several different labs, University of Iowa researchers have found a new genetic cause of the blinding eye disease retinitis pigmentosa (RP) and, in the process, discovered an entirely new version of the message that codes for the affected protein.

10 Aug 2011

Lentiviral vector can kill HIV-infected cells

In what represents an important step toward curing HIV, a USC scientist has created a virus that hunts down HIV-infected cells.

9 Aug 2011

De novo mutations account for 50% of sporadic cases of schizophrenia

Columbia University Medical Center researchers have shown that new, or "de novo," protein-altering mutations—genetic errors that are present in patients but not in their parents—play a role in more than 50 percent of "sporadic" —i.e., not hereditary—cases of schizophrenia.

8 Aug 2011

U.S. physicians spend more time, money for interaction with health plans than Canadian doctors

Physicians in the United States spend nearly four times as much dealing with health insurers and payers compared with doctors in Canada. Most of the difference stems from the fact that Canadian physicians deal with a single payer, in contrast to the multiple payers in the United States.

5 Aug 2011

Study finds HIV epidemics emerging in the Middle East and North Africa among gays

HIV epidemics are emerging in several countries in the Middle East and North Africa among men who have sex with men, a term that encompasses gay, non-gay identified homosexual men, and transgendered and bisexual men.

3 Aug 2011

Lipid nanocarriers in cancer diagnosis and therapy: New book

This book introduces fundamental principles of lipid nanocarriers science relevant to cancer therapy. The first in this field, it fills a need for an accurate, coherent and authoritative introduction to lipid nanocarriers focusing in cancer therapy; both because of the growing popularity of these modern drug delivery systems and also because of the emergent need of dealing with cancer treatment.

2 Aug 2011

New fluorescent tool can track mysterious workings of cellular RNAs

The ability to tag proteins with a green fluorescent light to watch how they behave inside cells so revolutionized the understanding of protein biology that it earned the scientific teams who developed the technique Nobel Prizes in 2008.

30 Jul 2011

EGT transfers clinical grade lentiviral vector to MSKCC

Errant Gene Therapeutics, LLC, a pioneering boutique drug development firm specializing in Rare Diseases, announced the transfer of its clinical grade lentiviral vector, TNS 9.55.3, to Memorial Sloan Kettering Cancer Center.

29 Jul 2011

PharmaNet to assist BrainStorm in obtaining FDA approval for ALS human clinical trials

BrainStorm Cell Therapeutics Inc., an innovative developer of adult stem cell technologies and CNS therapeutics, announced today that it has signed an agreement with PharmaNet LLC, an inVentiv Health Company, to assist in the development of the protocol and IND file for submission to the FDA for approval of BrainStorm's ALS human clinical trials in the United States.

26 Jul 2011

Promising drug for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a severely debilitating and lethal condition that affects 1 in 3,000. Females and males are affected, though females are rarely affected and are more often carriers. It is caused due to a defective gene that normally codes for the protein dystrophin, which helps provide a protective membrane around muscle fibers. Without this skin, muscle fibers become damaged and eventually die.

25 Jul 2011

FDA approves U.S. launch of Wiskott-Aldrich gene therapy clinical trial

The U.S. Food & Drug Administration (FDA) approved the launching in the U.S. of a clinical trial for gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome.

22 Jul 2011

NIAID awards $36M grant to BIDMC for AIDS vaccine research in nonhuman primates

Beth Israel Deaconess Medical Center (BIDMC) has been awarded a grant from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, to lead a Consortium for AIDS Vaccine Research in Nonhuman Primates.

21 Jul 2011

Cell biologists identify infection mechanism of adenoviruses

Mucosal epithelia do not have any receptors on the outer membrane for the absorption of viruses like hepatitis C, herpes, the adenovirus or polio, and are thus well-protected against pathogenic germs. However, certain viruses, such as the human immunodeficiency virus HIV, still manage to enter the body via the mucous membrane.

21 Jul 2011

Localized one-time gene therapy might effectively protect against atherosclerosis

A one-dose method for delivering gene therapy into an arterial wall effectively protects the artery from developing atherosclerosis despite ongoing high blood cholesterol.

21 Jul 2011

Camel blood can help scientists fight against cancer

Nanobodies produced from camel blood have unique properties, which can be used in future drug development. New research published in Journal of Controlled Release confirms that camel blood can help scientists in the fight against cancer.

15 Jul 2011

NIH grants $70M over five years to help search for HIV cure

NIH announced on Monday it will provide $70 million over five years to three collaborations searching for an HIV/AIDS cure, making it "the largest single investment yet … into finding a way to rid the virus from the body or at least reduce levels to the point that infected people can stop taking anti-HIV drugs - which many researchers until recently viewed as a hopeless quest," ScienceInsider reports.

13 Jul 2011

New research strategy for eradicating HIV from the body

An international team led by researchers from the University of California, San Francisco and the nonprofit Vaccine and Gene Therapy Institute of Port St. Lucie, Fla., has received a major grant from the National Institutes of Health to develop a strategy to eradicate HIV from the body.

12 Jul 2011

Merck researchers to participate in two new collaborative efforts to study HIV latency

Merck, known as MSD outside the United States and Canada, today announced that company researchers will participate in two new collaborative efforts led by the prominent academic institutions of the University of North Carolina (UNC) Chapel Hill and the University of California San Francisco (UCSF) to develop new approaches towards eradicating HIV, the virus that causes AIDS.

12 Jul 2011

genOway acquires worldwide exclusive license for RMCE technology from bluebird bio for genetically modified rodents

genOway and bluebird bio report today genOway acquisition of an exclusive worldwide license for the RMCE technology (developed at the Massachusetts Institute of Technology) in the field of genetically modified rodents.

12 Jul 2011

NIAID awards $14 million a year for new HIV research

Three research teams focused on developing strategies that could help to rid the body of HIV are receiving grants totaling more than $14 million a year, for up to five years, the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health announced today.

11 Jul 2011