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.

Study demonstrates ways in which quality measurement from EHRs can be improved

Health care providers and hospitals are being offered up to $27 billion in federal financial incentives to use electronic health records (EHRs) in ways that demonstrably improve the quality of care. The incentives are based, in part, on the ability to electronically report clinical quality measures.

16 Jan 2013

Experimental vaccine elicits antibodies that can protect against Ebola virus infection

Researchers at the National Institutes of Health (NIH) and Oregon Health & Science University (OHSU) have found that an experimental vaccine elicits antibodies that can protect nonhuman primates from Ebola virus infection.

16 Jan 2013

Aggressive breast cancer’s metastasis molecular switch identified

Scientists at Weill Cornell Medical College have discovered the molecular switch that allows aggressive triple negative breast cancer cells to grow the amoeba-like protrusions they need to crawl away from a primary tumor and metastasize throughout the body. Their findings, published in Cancer Cell, suggest a novel approach for developing agents to treat cancer once it has spread.

15 Jan 2013

Bionic devices: an interview with Dr Rylie Green

Bionic devices are implants which replace biological functions which have been lost due to nerve damage. They use electrical signals to stimulate the remaining nerve cells following disease or injury. Although the term bionic was only coined in the late 1950s, the earliest bionic devices were cardiac pacemakers, developed in the early 1900s. However the first commercial implantable units were not available until the 1950s.

14 Jan 2013

Blocking TRIP-Br2 gene protects against obesity and insulin resistance

Scientists observed that blocking the expression of the gene TRIP-Br2 in mice protects them against obesity and insulin resistance. The study shows that the gene modulates fat storage by regulating energy expenditure and lipolysis, the process which transforms fat into lipids for the body's energy consumption.

11 Jan 2013

Saliva gland test shows promise for Parkinson's

New research suggests that testing a portion of a person's saliva gland may be a way to diagnose Parkinson's disease.

11 Jan 2013

SLU researchers to test new screening method for MPS disorders among infants

Researchers at Saint Louis University have received a five-year, $2.5 million National Institutes of Health grant to test a new method of screening newborns for a group of rare, but serious genetic disorders that affect approximately 200 babies born in the U.S., each year.

11 Jan 2013

EHR implementation with technical support can improve patient care in small physician practices: Study

The relationship between a physician practice's adoption of electronic health records (EHR) and quality improvements in patient care remains unclear.

7 Jan 2013

New rat model simulates traumatic or surgical muscle tissue loss in humans

Penetrating soft tissue injuries that may be caused by bullet wounds or motor vehicle accidents, or exposure to explosive devices in military settings, can cause muscle loss resulting in functional disability and cosmetic deformity. Efforts underway to develop tissue engineering solutions to repair and replace damaged and lost muscle will benefit greatly from the availability of robust animal models to test these innovative therapeutic strategies.

4 Jan 2013

Implementation of appropriateness criteria required for elective procedures

Many of the most common inpatient surgeries in the United States are performed electively. These surgeries are expected to significantly increase with the enactment of the Affordable Care Act.

27 Dec 2012

Narrow therapeutic window for Canavan disease

Gene therapy for Canavan disease slows brain atrophy and stabilizes patients’ clinical condition, show findings that highlight the need for very early diagnosis and intervention.

22 Dec 2012

Gene therapy shows promise for severe coronary artery disease

Diagnosed with severe coronary artery disease, a group of patients too ill for or not responding to other treatment options decided to take part in a clinical trial testing angiogenic gene therapy to help rebuild their damaged blood vessels.

21 Dec 2012

Novel virus-based gene therapy holds promise for Canavan disease

Research led by Paola Leone, PhD, of the University of Medicine and Dentistry of New Jersey-School of Osteopathic Medicine (UMDNJ-SOM), demonstrates the long term safety and benefit of a virus-based gene therapy that has been applied for the first time in a clinical setting.

21 Dec 2012

Two recent experimental treatments improve visual function in mouse models of retinitis pigmentosa

Two recent experimental treatments - one involving skin-derived induced pluripotent stem (iPS) cell grafts, the other gene therapy - have been shown to produce long-term improvement in visual function in mouse models of retinitis pigmentosa (RP), according to the Columbia University Medical Center (CUMC) scientists who led the studies. At present, there is no cure for RP, the most common form of inherited blindness.

20 Dec 2012

Scientists uncover TDP-43 protein as possible target for treatment of ALS, FTD

Neuroscientists at Georgetown University Medical Center say they have new evidence that challenges scientific dogma involving two fatal neurodegenerative diseases - amyotrophic lateral sclerosis (ALS), and frontotemporal dementia (FTD) - and, in the process, have uncovered a possible therapeutic target as a novel strategy to treat both disorders.

20 Dec 2012

AAV-based gene therapy cocktail may help extend lives of children with Canavan disease

Results of a clinical trial that began in 2001 show that a gene therapy cocktail conveyed into the brain by a molecular special delivery vehicle may help extend the lives of children with Canavan disease, a rare and fatal neurodegenerative disorder.

20 Dec 2012

FDA grants Orphan Drug designation to Milo Biotechnology's AAV1-FS344 inhibitor

Milo Biotechnology today announced its AAV1-FS344 has been granted Orphan Drug designation from the FDA's Office of Orphan Products Development for treatment of Becker and Duchenne muscular dystrophy. AAV1-FS344 is a gene therapy-delivered myostatin inhibitor that increases muscle strength.

12 Dec 2012

Activated astrocytes could play a harmful role in Alzheimer's disease

A research team composed of University of Kentucky researchers has published a paper which provides the first direct evidence that activated astrocytes could play a harmful role in Alzheimer's disease.

12 Dec 2012

Weill Cornell research team develops new experimental drug therapy to target DLBCL

Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin's lymphoma and the seventh most frequently diagnosed cancer. The most chemotherapy resistant form of DLBCL, called activated B-cell - DLBCL (ABC-DLBCL), remains a major therapeutic challenge.

11 Dec 2012

uniQure commences acute intermittent porphyria Phase I clinical trial

uniQure B.V., a leader in the field of human gene therapy, today announced the start of its Phase I clinical trial in acute intermittent porphyria (AIP) with the treatment of the first patient.

11 Dec 2012