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.

Wayne State receives U.S. patent allowance for vision restoration technology

Technology to restore vision through the use of a component of green algae developed by Dr. Zhou-Han Pan, professor and scientific director of the Ligon Research Center of Vision at the Kresge Eye Institute at Wayne State University, and licensed to RetroSense Therapeutics, a biotechnology company dedicated to developing gene therapy approaches to vision restoration, announced that the U.S. Patent and Trademark Office has issued a Notice of Allowance for U.S. patent application (No. 12/299,574).

6 Mar 2013

Study finds how to shut down Bcl6 in diffuse large B-cell lymphoma

The powerful master regulatory transcription factor called Bcl6 is key to the survival of a majority of aggressive lymphomas, which arise from the B-cells of the immune system. The protein has long been considered too complex to target with a drug since it is also crucial to the healthy functioning of many immune cells in the body, not just B cells gone bad.

4 Mar 2013

UPV/EHU team uses lipid nanoparticles to develop new formulations for drug delivery

At the Faculty of Pharmacy of the Basque Public University (UPV/EHU) the Pharmacokinetics, Nanotechnology and Gene Therapy research team is using nanotechnology to develop new formulations that can be applied to drugs and gene therapy.

1 Mar 2013

Leading cancer researcher, Dr. Lewis Cantley, named a winner of the inaugural Breakthrough Prize in Life Sciences

Dr. Lewis Cantley, a leading cancer researcher credited with discovering a family of enzymes fundamental to understanding cancer, was named a winner of the inaugural Breakthrough Prize in Life Sciences, the world’s richest academic prize for medicine and biology. The prize, which carries a $3 million cash award, recognizes excellence in research aimed at curing intractable diseases and human life.

22 Feb 2013

Florida Hospital Pepin Heart Institute, USF partner for novel gene therapy trial

Cardiovascular disease specialists at Florida Hospital Pepin Heart Institute and Dr. Kiran C. Patel Research Institute affiliated with the University of South Florida announced they have enrolled their first patients into a clinical trial testing a novel gene therapy for the treatment of heart failure after ischemic injury. The therapy may promote the regeneration of heart tissue by encouraging the body to deploy more stem cells to the injury site.

22 Feb 2013

Researchers use 3D printing and injectable gels to create bioengineered ears

Physicians at Weill Cornell Medical College and biomedical engineers at Cornell University have succeeded in building a facsimile of a living human ear that looks and acts like a natural ear. Researchers believe their bioengineering method will finally succeed in the long quest by scientists and physicians to provide normal looking "new" ears to thousands of children born with a congenital ear deformity.

22 Feb 2013

Microbubble-ultrasound method improves cardiac output after heart attack

Scientists from the Bonn University Hospital successfully tested a method in mice allowing the morphological and functional sequelae of a myocardial infarction to be reduced. Tiny gas bubbles are made to oscillate within the heart via focused ultrasound - this improves microcirculation and decreases the size of the scar tissue. The results show that the mice, following myocardial infarction, have improved cardiac output as a result of this method, as compared to untreated animals.

21 Feb 2013

Stanford University neurobiology professor wins Mortimer D. Sackler, M.D. Prize

Columbia University College of Physicians and Surgeons and Weill Cornell Medical College have announced that the Mortimer D. Sackler, M.D. Prize for Distinguished Achievement in Developmental Psychobiology has been awarded to Carla Shatz, PhD, the Sapp Family Provostial Professor in Neurobiology at Stanford University in Palo Alto, California.

20 Feb 2013

Tiny microscope can help scientists observe neuronal firings in the brain

If you want to read a mouse's mind, it takes some fluorescent protein and a tiny microscope implanted in the rodent's head.

20 Feb 2013

Insights into HIV-1 virion makeup could improve gene therapy

A discovery about how the HIV-1 virion packages genomic RNA may help researchers to improve the efficacy of gene therapy.

18 Feb 2013

New approach could make gene therapy dramatically more effective for HIV patients

A research team from Case Western Reserve University School of Medicine has discovered an approach that could make gene therapy dramatically more effective for patients.

16 Feb 2013

ASGCT to honor UNC genetics professor with 2013 Outstanding New Investigator Award

Aravind Asokan, PhD, assistant professor of genetics in the University of North Carolina School of Medicine, was selected by the American Society of Gene and Cell Therapy to receive a 2013 Outstanding New Investigator Award.

15 Feb 2013

Ability to tune gene activity will permit refined research for drug resistance, cancer

Scientists who built a synthetic gene circuit that allowed for the precise tuning of a gene's expression in yeast have now refined this new research tool to work in human cells, according to research published online in Nature Communications.

13 Feb 2013

UPC develops new system for electroporation of cell cultures

Researchers from the Polytechnic University of Catalonia have developed a technique that improves and cuts the cost of a technique called electroporation, which involves opening pores in cell membranes using an electric field to introduce substances like drugs and DNA. Current methods are aggressive and expensive whereas the new system manages to apply low voltage electroporation with a small printed circuit board, which costs less than a Euro per unit and does not damage cells.

11 Feb 2013

Single gene therapy session can cure diabetes

Researchers from the Universitat Aut-noma de Barcelona (UAB), led by F-tima Bosch, have shown for the first time that it is possible to cure diabetes in large animals with a single session of gene therapy. As published this week in Diabetes, the principal journal for research on the disease, after a single gene therapy session, the dogs recover their health and no longer show symptoms of the disease.

10 Feb 2013

Two new methods could accelerate development of improved antisense drugs

Powerful antisense drugs that target disease-associated genes to block their expression can be used to treat a broad range of diseases. Though antisense therapy has been proven effective, challenges remain in ensuring that the drugs reach their intended targets.

2 Feb 2013

Weill Cornell and NewYork-Presbyterian create pioneering Institute for Precision Medicine

Recognizing that medicine is not "one size fits all," Weill Cornell Medical College and NewYork-Presbyterian Hospital have created the pioneering Institute for Precision Medicine at Weill Cornell and NewYork-Presbyterian/Weill Cornell Medical Center. This new, cutting-edge translational medicine research hub will explore the new frontier of precision medicine, offering optimal targeted, individualized treatment based on each patient's genetic profile.

1 Feb 2013

Insight into reasons why precocious puberty occurs in females

New research from Oregon Health & Science University has provided significant insight into the reasons why early-onset puberty occurs in females.

30 Jan 2013

New study may help treat patients with multiple myeloma

A study led by Robert G. Hawley, Ph.D., professor and chair of the department of anatomy and regenerative biology at the George Washington University (GW) School of Medicine and Health Sciences (SMHS), may help predict which patients with multiple myeloma will respond better to certain treatments.

25 Jan 2013

Researchers initiate first gene therapy clinical trial for Fabry disease

Researchers in Calgary have launched the first gene therapy clinical trial in the world for Fabry disease, a rare inherited enzyme deficiency that can shorten the lifespan of people who have it by as much as 40 years.

24 Jan 2013

Gene Therapy News and Research

Further Reading

Wayne State receives U.S. patent allowance for vision restoration technology

Study finds how to shut down Bcl6 in diffuse large B-cell lymphoma

UPV/EHU team uses lipid nanoparticles to develop new formulations for drug delivery

Leading cancer researcher, Dr. Lewis Cantley, named a winner of the inaugural Breakthrough Prize in Life Sciences

Florida Hospital Pepin Heart Institute, USF partner for novel gene therapy trial

Researchers use 3D printing and injectable gels to create bioengineered ears

Microbubble-ultrasound method improves cardiac output after heart attack

Stanford University neurobiology professor wins Mortimer D. Sackler, M.D. Prize

Tiny microscope can help scientists observe neuronal firings in the brain

Insights into HIV-1 virion makeup could improve gene therapy

New approach could make gene therapy dramatically more effective for HIV patients

ASGCT to honor UNC genetics professor with 2013 Outstanding New Investigator Award

Ability to tune gene activity will permit refined research for drug resistance, cancer

UPC develops new system for electroporation of cell cultures

Single gene therapy session can cure diabetes

Two new methods could accelerate development of improved antisense drugs

Weill Cornell and NewYork-Presbyterian create pioneering Institute for Precision Medicine

Insight into reasons why precocious puberty occurs in females

New study may help treat patients with multiple myeloma

Researchers initiate first gene therapy clinical trial for Fabry disease

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