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 gene therapy product reduces plasma cholesterol levels

Amsterdam Molecular Therapeutics (AMT) Holding N.V. a leader in the development of gene based therapies, today announced that its gene therapy product incorporating siRNA sequences into microRNA scaffolds to silence Apolipoprotein B100 (AAV-miApoB) was able to significantly lower plasma cholesterol levels in vivo over a period of 18 weeks. These preliminary results suggest that this approach could lead to a treatment for high cholesterol in humans

25 Oct 2010

Inovio announces publication of minimally-invasive intradermal device in journal Gene Therapy

Inovio Pharmaceuticals, Inc. a leader in the development of therapeutic and preventive vaccines against cancers and infectious diseases, announced today that a peer-reviewed research article describing the development of a new intradermal, minimally-invasive DNA vaccine delivery device was published in the prestigious journal Gene Therapy.

25 Oct 2010

NIH awards $3.9M for Fanconi anemia research

The National Institutes of Health has awarded a three-year, $3.9 million grant to Children's Hospital Boston researchers and their colleagues to develop a therapy to treat Fanconi anemia, a fatal genetic blood disease.

25 Oct 2010

Antigene therapy uses light energy to silence gene activity: Review article

Antigene therapy is a promising new treatment strategy that uses a DNA-based drug to pinpoint light energy to a target gene shutting down its activity.

22 Oct 2010

Researchers suggest gene therapy may treat patients with major depression

In a report published in the Oct. 20 issue of Science Translational Medicine, researchers at NewYork-Presbyterian Hospital/Weill Cornell Medical Center say animal and human data suggest gene therapy to the brain may be able to treat patients with major depression who do not respond to traditional drug treatment.

22 Oct 2010

Ceregene commences enrollment in CERE-120 Phase 2b study for Parkinson's disease

Ceregene, Inc., a biopharmaceutical company, announced today that enrollment has begun in a new double-blind sham surgery-controlled Phase 2b study evaluating CERE-120 in Parkinson's disease patients. CERE-120 is a gene therapy product that delivers the neurotrophic factor neurturin to degenerating or dying dopamine neurons. Data from the first Phase 2 clinical trial of CERE-120 are being published later this year in Lancet Neurology (to appear online October 21, 2010).

20 Oct 2010

Pharmacy graduate awarded national recognition for pioneering research in genetics

A young pharmacy graduate has been awarded national recognition for her pioneering research in the field of genetics which has the potential to treat diseases such as cancer and Parkinson's.

19 Oct 2010

Major advance in treatment of brain disorders with external genes

In the October 17 issue of Nature Methods, researchers at Thomas Jefferson University describe how they can transfer genes into brain neurons intravenously, using a viral gene delivery vehicle (vector) that causes no side effects.

18 Oct 2010

Researchers discover blocking beta-arrestin 1 protein can reduce severity of heart failure

A Nova Southeastern University (NSU) researcher has announced a breakthrough discovery to block a protein that can contribute to heart failure. His discovery will appear in an upcoming issue of the prestigious medical journal, the Journal of the American College of Cardiology.

18 Oct 2010

Psychological and social challenges faced by parents of children with undiagnosed learning disorders

Parents of children with undiagnosed learning disorders, developmental deficits, and congenital abnormalities face a host of psychological and social challenges, which are explored in detail in a reflective article in Genetic Testing and Molecular Biomarkers, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com).

14 Oct 2010

QIAGEN, Abbott enter agreement to strengthen testing menus for automated in-vitro diagnostic applications

QIAGEN and Abbott today announced that they have entered into an agreement that significantly strengthens both companies' testing menus for automated in-vitro diagnostic applications in the United States and Canada. Financial terms were not disclosed.

From QIAGEN Manchester Limited 13 Oct 2010

Health Robotics enters distribution agreements with Newico and Apta Medica for 7 new European countries

Health Robotics today announced that it recently signed exclusive distribution agreements for its CytoCare, i.v.STATION, and i.v.SOFT world-leading technologies in seven new European countries with regional industry leaders Newico and Apta Medica, rapidly continuing on its worldwide quest to pervasively provide safe and cost-effective Intravenous (IV) Admixture Compounding Automation to hospitals all over the world.

11 Oct 2010

Cincinnati Children's Hospital Medical Center named Centers of Excellence for Molecular Hematology

Cincinnati Children's Hospital Medical Center has been named one of five national Centers of Excellence for Molecular Hematology to find new gene and cell therapies for inherited diseases affecting blood cells.

8 Oct 2010

New reference material to standardize gene therapy protocols in clinical trials

The introduction of a new, fully characterized viral vector for use as reference material to help standardize gene therapy protocols in research applications and human clinical trials is described in an article in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com). The article, which is published online ahead of print, is available free online at www.liebertpub.com/hum

7 Oct 2010

Immune reaction to dystrophin may contribute to muscle disease: Study

An immune reaction to dystrophin, the muscle protein that is defective in patients with Duchenne muscular dystrophy, may pose a new challenge to strengthening muscles of patients with this disease, suggests a new study appearing in the October 7, 2010, issue of The New England Journal of Medicine.

7 Oct 2010

Patients with muscular dystrophy mount immune response to dystophin protein prior to gene therapy: Study

A clinical trial designed to replace the genetic defect causing the most common form of muscular dystrophy has uncovered an unexpected aspect of the disease. The trial, based on therapy designed by scientists at the University of North Carolina at Chapel Hill School of Medicine, showed that some patients mount an immune response to the dystrophin protein even before they have received the gene therapy.

7 Oct 2010

Study demonstrates potential of nontoxic envelope proteins as an alternative to pseudotype lentiviral vectors

Therapeutic lentiviral vectors are emerging as vital tools for molecular medicine as evidenced by the growing number of clinical trials using these vector systems. From a basic research standpoint, lentiviral vectors are very intriguing substrates. On the one hand, the HIV-1 genome offers expanded cloning capacity and the capability to transduce nondividing cells such as hematopoietic stem cells (HSCs) and neurons.

4 Oct 2010

Encorium announces extension of expiration date for pending rights offering

Encorium Group, Inc. , a full service multinational clinical research organization (CRO) conducting studies in over 30 countries for many of the world's leading pharmaceutical and biotechnology companies, today announced that it has extended the expiration date for its pending rights offering from September 30, 2010 to October 15, 2010. The subscription rights may now be exercised at any time prior to 5:00 p.m. ET on Friday, October 15, 2010.

1 Oct 2010

NIH awards three grants to explore novel treatment strategies for muscular dystrophy

Three grants totaling more than $4.5 million, from agencies of the National Institutes of Health, will be used to explore novel treatment strategies for muscular dystrophy.

30 Sep 2010

CAP cells produce high virus titers upon influenza strain infection

CEVEC Pharmaceuticals, the developer of an extremely potent protein expression system based on human CAP cells, announced today that CAP cells produce high virus titers upon infection with influenza strains. The virus titers are as good as or even higher than with MDCK and Vero cells, host cells currently used for influenza vaccine production.

29 Sep 2010

Gene Therapy News and Research

Further Reading

New gene therapy product reduces plasma cholesterol levels

Inovio announces publication of minimally-invasive intradermal device in journal Gene Therapy

NIH awards $3.9M for Fanconi anemia research

Antigene therapy uses light energy to silence gene activity: Review article

Researchers suggest gene therapy may treat patients with major depression

Ceregene commences enrollment in CERE-120 Phase 2b study for Parkinson's disease

Pharmacy graduate awarded national recognition for pioneering research in genetics

Major advance in treatment of brain disorders with external genes

Researchers discover blocking beta-arrestin 1 protein can reduce severity of heart failure

Psychological and social challenges faced by parents of children with undiagnosed learning disorders

QIAGEN, Abbott enter agreement to strengthen testing menus for automated in-vitro diagnostic applications

Health Robotics enters distribution agreements with Newico and Apta Medica for 7 new European countries

Cincinnati Children's Hospital Medical Center named Centers of Excellence for Molecular Hematology

New reference material to standardize gene therapy protocols in clinical trials

Immune reaction to dystrophin may contribute to muscle disease: Study

Patients with muscular dystrophy mount immune response to dystophin protein prior to gene therapy: Study

Study demonstrates potential of nontoxic envelope proteins as an alternative to pseudotype lentiviral vectors

Encorium announces extension of expiration date for pending rights offering

NIH awards three grants to explore novel treatment strategies for muscular dystrophy

CAP cells produce high virus titers upon influenza strain infection

Achieving and maintaining ISO 27001 and BS 10012 certifications

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

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