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.

Biokine, BioLineRx sign license agreement to develop and commercialize BL-8040 for AML

BioLineRx Ltd., a biopharmaceutical drug development company, announced today that it has signed an exclusive, worldwide license agreement with Biokine Therapeutics Ltd., a Clal Biotechnology Industries portfolio company, for the development and commercialization of BL-8040 (formerly BKT-140), a Phase II ready drug candidate for the treatment of acute myeloid leukemia (AML), as well as other types of hematological cancer.

4 Sep 2012

Gene therapy approach restores ability to smell in mouse model of congenital anosmia

Scientists funded by the National Institutes of Health have restored the ability to smell in a mouse model of a human genetic disorder that causes congenital anosmia-the inability to smell from birth. The approach uses gene therapy to regrow cilia, cell structures that are essential for olfactory function.

3 Sep 2012

Scientists restore sense of smell in mice through gene therapy

Scientists have restored the sense of smell in mice through gene therapy for the first time -- a hopeful sign for people who can't smell anything from birth or lose it due to disease.

3 Sep 2012

Glaucoma and cell death: an interview with Dr. Vivek Gupta

Glaucoma is normally caused by increased pressure of aqueous humour fluid in the eye. A defining feature of this disease is destruction of a particular type of cells in the retina called retinal ganglion cells and excavation of the optic disc region.

31 Aug 2012

Weill Cornell Medical College receives three new research grants for translational blood cancer study

Weill Cornell Medical College has been awarded three new research grants from the Leukemia & Lymphoma Society totaling $1.8 million. The funds will support critically-needed translational research for blood cancers, accelerating promising discoveries from the laboratory to the patient's bedside.

31 Aug 2012

SUMO1 protein may treat and/or prevent heart failure

A promising new drug target for the treatment and prevention of heart failure has been discovered by researchers at Mount Sinai School of Medicine in New York, NY, US. The study was presented at the ESC Congress 2012 by principal investigator Professor Roger J. Hajjar, MD.

26 Aug 2012

NCKU research team identifies Au nanorod complex for cancer therapy

A National Cheng Kung University (NCKU) research team has discovered Near-Infrared Light-Responsive oligonucleotide-gated Au nanoensembles (Au nanorod complex), a potent new anti-cancer complex that is seen as a promising targeted therapy for curing cancer.

24 Aug 2012

SSEA4+ marker helps identify and isolate precursors of insulin-producing pancreatic cells

For the millions of people worldwide with type 1 diabetes who cannot produce sufficient insulin, the potential to transplant insulin-producing cells could offer hope for a long-term cure. The discovery of a marker to help identify and isolate stem cells that can develop into insulin-producing cells in the pancreas would be a critical step forward and is described in an article in BioResearch Open Access, a new bimonthly peer-reviewed open access journal from Mary Ann Liebert, Inc.

22 Aug 2012

Gene therapy applied to peripheral arterial disease

Hepatocyte growth factor gene therapy could be used to treat patients with severe peripheral arterial disease, suggest results of a phase I clinical trial.

21 Aug 2012

Novel device provides retina's neural code to restore normal vision

Two researchers at Weill Cornell Medical College have deciphered a mouse's retina's neural code and coupled this information to a novel prosthetic device to restore sight to blind mice.

15 Aug 2012

Combination of novel drug therapy and PSA test can effectively detect prostate cancer

Researchers have successfully developed and tested a new prostate cancer screening method that uses the combined power of a novel drug therapy and changes in PSA levels over time to identify men with a high PSA who are more likely to have aggressive prostate cancer despite negative biopsies.

12 Aug 2012

Researchers develop and test new prostate cancer screening method

9 Aug 2012

Weill Cornell Medical College receives NCATS grant to fund Clinical and Translational Science Center

Weill Cornell Medical College has received $49.6 million from the National Center for Advancing Translational Sciences (NCATS), of the National Institutes of Health (NIH), to fund its Clinical and Translational Science Center (CTSC).

3 Aug 2012

Researchers discover genetic process that can restore function to a CF defective protein

Researchers at the University of Iowa Carver College of Medicine have discovered a genetic process that can restore function to a defective protein, which is the most common cause of cystic fibrosis (CF).

2 Aug 2012

Chk1 gene alone is sufficient to kill cancer cells

Researchers at Case Western Reserve University School of Medicine have discovered a mutant form of the gene, Chk1, that when expressed in cancer cells, permanently stopped their proliferation and caused cell death without the addition of any chemotherapeutic drugs.

1 Aug 2012

Obesity also affects sexual and urinary health in men

As a man's waistline grows, so can his experience with sexual dysfunction and frequent urination, say researchers at NewYork-Presbyterian Hospital/Weill Cornell Medical Center. The study, published in the August issue of the British Journal of Urology International (BJUI), is the first to comprehensively show that obesity in men affects not just their hearts and metabolism, but also their sexual and urinary health.

31 Jul 2012

Australia's top medical researchers for 2012 announced

Finalists for one of Australia’s longest running and most prestigious awards for medical research were announced today. This year’s winner will join a list of alumni winners that includes many of the country’s most respected scientists.

31 Jul 2012

Researchers isolate elusive human gene that causes NMNAT1-related Leber congenital amaurosis

Researchers from the Massachusetts Eye and Ear Infirmary, The Children's Hospital of Philadelphia, Loyola University Chicago Health Sciences Division and their collaborators have isolated an elusive human gene that causes a common form of Leber congenital amaurosis (LCA), a relatively rare but devastating form of early-onset blindness. The new LCA gene is called NMNAT1.

30 Jul 2012

Cedars-Sinai to develop stem cell treatments for patients with ALS

Cedars-Sinai Regenerative Medicine Institute today was awarded a $17.8 million grant from the California Institute for Regenerative Medicine to develop stem cell treatments for patients with ALS, also known as Lou Gehrig's Disease, a progressive, fatal and currently incurable neurodegenerative disease.