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.

Scientists reveal inside story of European regulatory review and approval of Glybera

The scientists who led the team that developed Glybera, the first gene therapy drug approved for use in the Western world, provide a fascinating first-person account of their pioneering work in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

16 May 2013

Inhibiting powerful protein with new agents may supply broad benefit for lymphoma patients

A soon-to-be-tested class of drug inhibitors were predicted to help a limited number of patients with B-cell lymphomas with mutations affecting the EZH2 protein.

14 May 2013

Researchers successfully test new anti-cocaine vaccine in primates

Researchers at Weill Cornell Medical College have successfully tested their novel anti-cocaine vaccine in primates, bringing them closer to launching human clinical trials.

13 May 2013

UNM Cancer Center to participate in Phase 3 clinical trial of novel treatment for men with prostate cancer

The University of New Mexico Cancer Center is among a few select institutions nationwide participating in a Phase 3 clinical trial studying a novel treatment for men with newly diagnosed, localized prostate cancer.

13 May 2013

Cell Medica treats first pediatric patient in ASPIRE Trial

Cell Medica today announced the treatment of the first patient in the ASPIRE Trial, an early stage Phase I/II clinical study investigating the safety and efficacy of Cytovir ADV for the treatment of adenovirus infections in immunosuppressed pediatric patients following bone marrow transplantation.

8 May 2013

Neuroperspective reviews status and prospects of therapeutics for Parkinson's disease

NI Research, the leading publisher of independent research on the neurotherapeutics industry, has released the May issue of NeuroPerspective, which reviews the status and prospects of therapeutics for Parkinson's.

7 May 2013

Researchers identify how a defective protein plays central role in Giant Axonal Neuropathy

A team of international researchers led by Northwestern Medicine scientists has identified how a defective protein plays a central role in a rare, lethal childhood disease known as Giant Axonal Neuropathy, or GAN.

29 Apr 2013

Researchers discover gene that can cause 3 different diseases depending on its alteration

An international research consortium led by the Universitat Aut-noma de Barcelona (UAB), the CIBERER and the University of Wurzburg (Germany) has discovered a gene that can cause three totally different diseases, depending on how it is altered.

26 Apr 2013

Sofosbuvir is safer than interferon for hepatitis C patients, say scientists

A new drug is offering dramatic cure rates for hepatitis C patients with two subtypes of the infection -- genotype 2 and 3, say a team of scientists led by Weill Cornell Medical College researchers.

25 Apr 2013

Tissue engineering combined with gene therapy can manage repair of defective articular cartilage

Researchers at Micro Orthopaedics, Zhongnan Hospital of Wuhan University, led by Dr. Ai-xi Yu, have suggested that articular cartilage defects can be repaired by a novel thermo-sensitive injectable hydrogel engineered with gene modified bone marrow mesenchymal stromal cells.

24 Apr 2013

Methodist Hospital will soon have new brain and pituitary tumor treatment center

Brain and pituitary tumor patients will soon have access to a state-of-the-art facility at The Methodist Hospital, a result of a $10 million dollar commitment from a Houston oil executive.

24 Apr 2013

Animal models of Huntington's disease allow better insights into disease's genetics

Scientific progress in Huntington's disease (HD) relies upon the availability of appropriate animal models that enable insights into the disease's genetics and/or pathophysiology.

23 Apr 2013

Xenon Pharmaceuticalsn receives BIOTECanada's Gold Leaf Company of the Year Award for 2013

Xenon Pharmaceuticals Inc. is pleased to announce that it has been awarded BIOTECanada's Gold Leaf Company of the Year Award for 2013.

22 Apr 2013

Penn researcher wins Distinguished Clinical Research Achievement Award for innovations in gene therapy

A gene therapy study focused on finding a cure for a rare congenital blinding disease has been recognized as one of the ten most outstanding clinical research projects of the year by the Clinical Research Forum (CRF).

22 Apr 2013

Ceregene announces top-line data from CERE-12 Phase 2b clinical study for Parkinson's disease

Ceregene, Inc. today announced the top-line data from its double-blind, randomized, controlled Phase 2b clinical study of CERE-120 (AAV-neurturin), a gene therapy product designed to deliver the neurotrophic factor neurturin, for Parkinson's disease.

20 Apr 2013

Penn Medicine gene therapy study receives Distinguished Clinical Research Achievement Award

20 Apr 2013

Tacere Therapeutics submits TT-034 clinical trial application to Recombinant DNA Advisory Committee

RNAi-based therapeutics company Benitec Biopharma Limited today announced that the company's wholly owned US subsidiary, Tacere Therapeutics Inc., has submitted an application to the US National Institutes of Health's Recombinant DNA Advisory Committee.

18 Apr 2013

Death of heart tissue could be prevented with new medicines, say TUSM scientists

A major factor in the advance of heart disease is the death of heart tissue, a process that a team of scientists at Temple University School of Medicine's Center for Translational Medicine think could be prevented with new medicines.

15 Apr 2013

New combination therapy could help restore vision

Researchers have discovered that using two kinds of therapy in tandem may be a knockout combo against inherited disorders that cause blindness. While their study focused on man's best friend, the treatment could help restore vision in people, too.

10 Apr 2013

Weill Cornell Medical College sets up new Feil Family Brain and Mind Research Institute

Weill Cornell Medical College has established the new Feil Family Brain and Mind Research Institute, a unique, multidisciplinary translational neuroscience research hub. Named in honor of long-time benefactors Gertrude and Louis Feil, the institute was created with a generous $28 million gift from the Feil Family.

9 Apr 2013