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 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

Study shows how tafamidis drug works against amyloid disease

Scientists from The Scripps Research Institute and Pfizer Inc. have published a new study showing how a new drug called tafamidis (Vyndaqel) works.

31 May 2012

Gene therapy can effectively treat ADA-deficient severe combined immunodeficiency

Severe combined immunodeficiency is defect in the immune system that results in a loss of the adaptive immune cells known as B cells and T cells. Mutations in several different genes can lead to the development of severe combined immunodeficiency, including mutation of the adenosine deaminase (ADA) gene. Traditional treatment options, such as enzyme replacement therapy, are of limited efficacy, but bone marrow transplant from a compatible donor leads to a better response.

25 May 2012

Optogenetic Therapies for Vision workshop to be held on June 1

The Foundation Fighting Blindness and Massachusetts Eye and Ear will host the Optogenetic Therapies for Vision workshop on Friday, June 1, from 8 a.m.-6 p.m., in Boston.

24 May 2012

Molecular Therapy releases characteristics and activity of SNS01 formulation

Senesco Technologies, Inc., announced today that the characteristics and activity of SNS01 have been released on-line in Molecular Therapy, the official journal of the American Society for Gene & Cell Therapy.

24 May 2012

Study shows how chromosomal mutation likely contributes to early development of prostate cancer

Up to half of all prostate cancer cells have a chromosomal rearrangement that results in a new "fusion" gene and formation of its unique protein -- but no one has known how that alteration promotes cancer growth. Now, Weill Cornell Medical College researchers have found that in these cancer cells, the 3-D architecture of DNA, wrapped up in a little ball known as a chromatin, is warped in such a way that a switch has been thrown on thousands of genes, turning them on or off to promote abnormal, unchecked growth. Researchers also found that new chromosomal translocations form, further destabilizing the genome.

23 May 2012

IntelliCell enters sponsored research agreement with ICERM

IntelliCell BioSciences, Inc. announced today that it has entered a sponsored research agreement with the Institute for Cell Engineering and Regenerative Medicine (ICERM) at the University of Florida.

23 May 2012

Gene Therapy News and Research

Further Reading

New type of micromachine can penetrate tissue and overcome cellular barriers

Targeted delivery of therapeutic gene can effectively treat Pompe disease

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

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

NewYork-Presbyterian/WCMC join new MDS Clinical Research Consortium

Researchers unlock structure of STING protein

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

Single-dose vaccine for treatment of cocaine addiction

Implanted device delivers genetically engineered neurotrophic factor directly to the brain

Researchers develop double viral vector transfection technique

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

Scientists discover new function for thrombospondin protein

VGXI, OncoSec partner to produce interleukin-12 plasmid DNA

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

Study shows how tafamidis drug works against amyloid disease

Gene therapy can effectively treat ADA-deficient severe combined immunodeficiency

Optogenetic Therapies for Vision workshop to be held on June 1

Molecular Therapy releases characteristics and activity of SNS01 formulation

Study shows how chromosomal mutation likely contributes to early development of prostate cancer

IntelliCell enters sponsored research agreement with ICERM

Ultimate guide to the essential tools for the characterization of proteins, polymers and nanoparticles eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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