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.

Single dose breast cancer treatment helps forego 6-7 week radiation regimen

NewYork-Presbyterian Hospital/Weill Cornell Medical Center is the first and only hospital in the five boroughs of New York City to offer women with early stage breast cancer INTRABEAM radiotherapy, an innovative radiation treatment delivered in a single dose at the time of surgery.

5 Jul 2012

Nicotine addiction: An interview with Dr. Crystal

Cigarette smoking and nicotine addiction are widespread problems throughout the world. Many people continue to smoke despite high taxes and the available public information on the dangers of smoking.

3 Jul 2012

Treating Sickle Cell Disease with L-Glutamine

By focusing on how best to help patients, researchers can sometimes find solutions that are much simpler than anticipated. That has been the guiding approach to our efforts to develop a treatment for sickle cell disease.

3 Jul 2012

Safer alternative to experimental gene therapy against HIV infection

Scientists at The Scripps Research Institute have discovered a surprisingly simple and safe method to disrupt specific genes within cells. The scientists highlighted the medical potential of the new technique by demonstrating its use as a safer alternative to an experimental gene therapy against HIV infection.

2 Jul 2012

Vaccine for nicotine addiction developed

Researchers at Weill Cornell Medical College have developed and successfully tested in mice an innovative vaccine to treat nicotine addiction.

28 Jun 2012

Gene transfer technique eliminates specific neural circuit linked to Parkinson's disease

In the brains of humans and non-human primates, over 100 billion nerve cells build up complicated neural circuits and produce higher brain functions. When an attempt is made to perform gene therapy for neurological diseases like Parkinson's disease, it is necessary to specify a responsible neural circuit out of many complicated circuits.

27 Jun 2012

New type of micromachine can penetrate tissue and overcome cellular barriers

Microscopically small submarines that can swim through our blood to clear out clogged arteries or destroy malignant tumors. This concept may sound utopian, but it isn’t. Various micro- and nanomachines have in fact already been developed.

27 Jun 2012

Targeted delivery of therapeutic gene can effectively treat Pompe disease

Gene therapy to replace the protein missing in Pompe disease can be effective if the patient's immune system does not react against the therapy. Targeted delivery of the gene to the liver, instead of throughout the body,suppresses the immune response, improving the therapeutic effect, according to an article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc.

26 Jun 2012

WCMC researchers devise innovative boxer-like strategy to fight multiple myeloma

Weill Cornell Medical College researchers have devised an innovative boxer-like strategy, based on the serial use of two anti-cancer drugs, to deliver a one-two punch to first weaken the defenses of multiple myeloma and then deliver the final knock-out punch to win the fight.

23 Jun 2012

Gold nanoparticles with slight positive charge work collectively to unravel DNA's double helix

New research from North Carolina State University finds that gold nanoparticles with a slight positive charge work collectively to unravel DNA's double helix. This finding has ramifications for gene therapy research and the emerging field of DNA-based electronics.

21 Jun 2012

NewYork-Presbyterian/WCMC join new MDS Clinical Research Consortium

NewYork-Presbyterian/Weill Cornell Medical Center is one of six institutions selected to join the newly-founded MDS Clinical Research Consortium.

20 Jun 2012

Researchers unlock structure of STING protein

Researchers at Weill Cornell Medical College have unlocked the structure of a key protein that, when sensing certain viruses and bacteria, triggers the body's immediate immune response.

20 Jun 2012

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD).

19 Jun 2012

Single-dose vaccine for treatment of cocaine addiction

A single-dose vaccine capable of providing immunity against the effects of cocaine offers a novel and groundbreaking strategy for treating cocaine addiction is described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc.

19 Jun 2012

Implanted device delivers genetically engineered neurotrophic factor directly to the brain

Studies suggest that neurotrophic factors, which play a role in the development and survival of neurons, have significant therapeutic and restorative potential for neurologic diseases such as Huntington's disease. However, clinical applications are limited because these proteins cannot easily cross the blood brain barrier, have a short half-life, and cause serious side effects.

19 Jun 2012

Researchers develop double viral vector transfection technique

The collaborative research team led by Professor Tadashi ISA, Project Assistant Professor Masaharu KINOSHITA from The National Institute for Physiological Sciences, The National Institutes of Natural Sciences and Fukushima Medical University and Kyoto University, developed "the double viral vector transfection technique" which can deliver genes to a specific neural circuit by combining two new kinds of gene transfer vectors.

18 Jun 2012

High doses of tiny milk vitamin produces numerous health benefits in mice

A novel form of vitamin B3 found in milk in small quantities produces remarkable health benefits in mice when high doses are administered, according to a new study conducted by researchers at Weill Cornell Medical College and the Polytechnic School in Lausanne, Switzerland.

15 Jun 2012

Scientists discover new function for thrombospondin protein

Scientists have discovered a new function for a protein that protects cells during injury and could eventually translate into treatment for conditions ranging from cardiovascular disease to Alzheimer's.

8 Jun 2012

VGXI, OncoSec partner to produce interleukin-12 plasmid DNA

OncoSec Medical Inc., which is developing its advanced-stage OncoSec Medical System (OMS) ElectroOncology therapies to treat solid tumors, announced today that the Company has entered into an exclusive cGMP manufacturing agreement with VGXI Inc. (VGXI) for the production of interleukin-12 plasmid DNA.

6 Jun 2012

Chatham, Baxter partner to develop and commercialize hemophilia B gene therapy treatment

Baxter International Inc. today announced that it has entered into an exclusive global agreement with Chatham Therapeutics, LLC, an affiliate of Asklepios BioPharmaceutical, Inc. (AskBio), for the development and commercialization of potential treatments for hemophilia B utilizing Chatham's gene therapy technology.

From Baxter International Inc. 5 Jun 2012