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.

Orphan Disease Center establishes new initiative that focuses on ALS

The Orphan Disease Center in the Perelman School of Medicine at the University of Pennsylvania has established a new Program of Excellence for Motor Neuron Disease.

10 Jan 2017

New research suggests novel combination approach to fight against gliomas

"Devastating" and "dismal." That's how leading researchers describe the present outlook for malignant brain tumors. The median survival rate for patients with glioblastoma multiforme, or GBM, is a mere 14.2 months.

4 Jan 2017

AAV-based gene therapy to treat liver disorders advances into human testing

Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing.

30 Dec 2016

Studies in non-human primates provide more predictive results in genetic causes of blindness

Many gene therapy-based approaches are in development to combat genetic and other causes of blindness and vision loss, and much can be learned about the safety and effectiveness of these promising new therapies by studying them first in non-human primates before initiating clinical trials, as shown by the results of a study published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

29 Dec 2016

Researchers use CRISPR-based genetic screening to identify three promising HIV treatment targets

Investigators from Whitehead Institute, the Ragon Institute of MGH, MIT and Harvard and the Broad Institute of MIT and Harvard have used CRISPR-Cas9 gene-editing technology to identify three promising new targets for treatment of HIV infection.

20 Dec 2016

URMC researchers identify potential new means of treating severe genetic diseases in children

University of Rochester Medical Center researchers believe they have identified a potential new means of treating some of the most severe genetic diseases of childhood, according to a study in PLOS Biology.

16 Dec 2016

Review highlights link between intestinal microbiota and colorectal cancer development

Recent evidence from animal models suggests a role for specific types of intestinal bacteria in the development of colorectal cancer (CRC).

9 Dec 2016

Hemophilia B patients produce near-normal levels of clotting factor IX after gene therapy, study shows

Researchers are reporting the highest and most sustained levels to date of the essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B.

4 Dec 2016

Combination of demineralized bone and gene therapy shows promise for treating pseudarthrosis

A team headed by Professor Albert Rizvanov, director of the Gene and Cell Technologies Open Lab, created a gene therapy drug that encodes growth factors for the stimulation of blood vessel and bone formation.

2 Dec 2016

BioCision reveal new company focussing on clinical technology development

BioCision, LLC, a life science research and development company that standardizes basic laboratory processes, today announced the formation of MedCision, Inc., an independent company focused on automation of pre-clinical and clinical processes.

1 Dec 2016

Penn researchers use CRISPR/Cas9 gene targeting approach to treat hemophilia B in mice

CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations.

1 Dec 2016

Researchers use microbubbles and ultrasound to transport drugs across blood–brain barrier

The impassable blood-brain barrier prevents microorganisms from entering our brain, however it also blocks medicines that could help treat Parkinson's, Alzheimer's and other neurodegenerative diseases.

30 Nov 2016

CNIC study characterizes key signal that impedes intercellular communication

A team of scientists at the Centro Nacional de Investigaciones Cardiovasculares Carlos III, led by Prof. Francisco Sánchez-Madrid, has characterized a cell signal that impedes intercellular communication and could play a central role in biomedical strategies such as gene therapy, vaccine design, and immunotherapy.

29 Nov 2016

BAG3 protein plays protective role by limiting reperfusion injury to the heart

The inability of cells to eliminate damaged proteins and organelles following the blockage of a coronary artery and its subsequent re-opening with angioplasty or medications - a sequence known as ischemia/reperfusion - often results in irreparable damage to the heart muscle.

17 Nov 2016

Novel approach combines imaging and electroanatomical mapping for targeted cardiac gene transfer

Gene therapy to repair damaged heart muscle is most likely to succeed if it can be injected at the site of ischemia where there is viable myocardium with reduced contractile ability, and a new technique that combines imaging and electroanatomical mapping does just that.

15 Nov 2016

Retinitis pigmentosa can be slowed by reprogramming metabolism of photoreceptors, study shows

Columbia University Medical Center researchers have demonstrated that vision loss associated with a form of retinitis pigmentosa (RP) can be slowed dramatically by reprogramming the metabolism of photoreceptors, or light sensors, in the retina.

15 Nov 2016

Researchers start enrolling patients for Phase II gene therapy trial for choroideremia

Following on from a successful world’s first Phase I gene therapy trial for choroideremia, Professor Robert MacLaren and his team at Oxford University and the Oxford Eye Hospital at the John Radcliffe Hospital have started a Phase II trial enrolling 30 patients.

11 Nov 2016

Researchers propose model photomotors for rapid nanoparticle transport

Scientists from the Moscow Institute of Physics and Technology, Semenov Institute of Chemical Physics of the Russian Academy of Sciences, and Chuiko Institute of Surface Chemistry of the National Academy of Sciences of Ukraine have proposed a model nanosized dipole photomotor based on the phenomenon of light-induced charge redistribution.

11 Nov 2016

Caltech researchers develop new technique that increases safety of TCR gene therapy

The human body produces T cells to recognize and fight disease. Each T cell has a unique T cell receptor (or TCR) on its surface that surveils small fragments of proteins presented by other cells.

8 Nov 2016

Single gene therapy for lipoprotein lipase deficiency reduces severity and frequency of pancreatitis

Over a 6-year period, patients with the genetic disease lipoprotein lipase deficiency (LPLD) who received a single gene therapy treatment of alipogene tiparvovec had a marked reduction in the severity and frequency of pancreatitis.