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.

Optoceutics technique opens up new horizons for regenerative medicine

Using light to facilitate the formation of new blood vessels: it is the breakthrough outcome of a research study carried out by researchers at Istituto Italiano di Tecnologia in Milan (Italy). The study was published in Science Advances.

30 Sep 2019

New CRISPR-Cas9 variant has potential to increase precision during gene therapy

Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase precision during gene therapy in humans.

30 Sep 2019

New trial shows improved survival for patients with advanced colorectal BRAF-mutant cancer

A clinical trial has shown that using a combination of three drugs that target a BRAF gene mutation in patients with mCRC effectively boosts overall survival.

30 Sep 2019

Johns Hopkins researchers develop new way to deliver sight-saving gene therapy to the retina

In experiments with rats, pigs and monkeys, Johns Hopkins Medicine researchers have developed a way to deliver sight-saving gene therapy to the retina.

From Johns Hopkins Medicine 30 Sep 2019

New NIH program aims to improve lives for people with inherited blood and immune diseases

People with inherited diseases of the blood and immune system can now receive treatment at the National Institutes of Health Clinical Center through a new, streamlined program that integrates expertise from many medical specialties and related basic science disciplines.

26 Sep 2019

Study shows how AF4-MALS-UV can separate and size large AAV aggregates

Postnova Analytics has published a new applications study that reports on how Asymmetric Flow Field Flow Fractionation (AF4) coupled with multi-angle light scattering (MALS) and Ultraviolet (UV) detectors can be used to quantify Adeno-Associated Virus (AAV) aggregation.

From Postnova Analytics 25 Sep 2019

7-year-old treated with FDA-approved gene therapy for Leber congenital amaurosis

When Zion Grant was 3 months old his mother Daisy Dodd noticed he wasn't tracking her with his eyes. Zion would fixate on a light in the room, but not on her face.

24 Sep 2019

Non-viral gene delivery method more effective and safer than standard viral approaches

The non-viral, bio-inspired gene delivery method developed by researchers at RMIT University has proven effective in laboratory tests and is safer than standard viral approaches.

24 Sep 2019

Scientists to present new findings on gene therapy at Neuroscience 2019

Gene therapy shows promise as a way to treat and cure presently incurable neurological diseases, including Alzheimer's disease, Parkinson's disease, and amyotrophic lateral sclerosis (ALS).

24 Sep 2019

Faster, cheaper, better: improved gene therapy tool production

The heart of modern therapy is increasingly personalized and targeted at specific cellular or genetic deficits. This requires gene therapy, in many cases. A new study, published on 19 September 2019 in the journal Nature, describes an improved way to produce purified gene carrier viruses called AAVs cheaply and quickly, from the culture medium.

22 Sep 2019

Novel gene therapy for glycogen storage disease shows positive results

At the Association for Glycogen Storage Disease's 41st Annual Conference, Dr. David Weinstein of UConn School of Medicine and Connecticut Children's presented his groundbreaking, one-year clinical trial results for the novel gene therapy treatment for glycogen storage disease.

20 Sep 2019

BUSM researchers develop advanced protocol to improve gene therapy tool production

A method to create a faster and lower cost alternative for a gene therapy tool has been developed by Boston University School of Medicine researchers.

19 Sep 2019

The resurgence of gene therapy pioneer

Twenty years ago, scientists and investors were giddy over the promise of gene therapy to cure a host of deadly diseases. But the tragic death of a young man from an experimental treatment in 1999 nearly ended a scientist's career and the entire field.

19 Sep 2019

Zylka lab receives NIH grant to advance treatments for neurodevelopmental disorders

The National Institutes of Health have awarded two separate grants totaling $6.1 million to Mark Zylka, PhD, director of the UNC Neuroscience Center. One of the grants was co-awarded to Ben Philpot, PhD, associate director of the center at the UNC School of Medicine.

18 Sep 2019

Patients with non-Hodgkin's lymphoma may benefit from radiation, experience reduced side effects

Treating non-Hodgkin's lymphoma patients with radiation therapy as an additional treatment while they wait for their CAR T cells to be manufactured may reduce the risk of CAR T therapy side effects once it is administered, according to a new study from researchers in the Abramson Cancer Center at the University of Pennsylvania.

18 Sep 2019

Children's Tumor Foundation harnesses interest in rare disease research through NF Innovation Week

Neurofibromatosis, a genetic disorder that causes tumors to grow on nerves throughout the body and which affects 1 in 3,000 people - that's over 2.5 million worldwide - has no cure and few workable treatment options.

15 Sep 2019

Nano-sized bubbles in our body could effectively fight cancer

Healthy cells in our body release nano-sized bubbles that transfer genetic material such as DNA and RNA to other cells. It's your DNA that stores the important information necessary for RNA to produce proteins and make sure they act accordingly.

15 Sep 2019

New gene therapy helps stroke patients develop new neurons

Ischemic stroke is a condition in which parts of the brain lose their blood supply, causing nerve damage. Once brain tissue suffers irreparable harm, the patient will experience irreversible disability or death, depending on the extent of neuronal loss.

12 Sep 2019

New approach enables the body to produce specific antibodies

Researchers at KU Leuven in Belgium have developed a technique to make sheep produce new antibodies simply by injecting the DNA building blocks.

12 Sep 2019

BIA Separations launches Cornerstone AAV Process Development Service to improve gene therapy production

BIA Separations, a leading bio-chromatography development and manufacturing company, today announced the introduction of CORNERSTONE AAV Process Development Service, a program of integrated process development services and novel technology designed to improve the robustness and yield of AAV-based gene therapy production, while improving the safety of therapeutic products.

12 Sep 2019

Gene Therapy News and Research

Further Reading

Optoceutics technique opens up new horizons for regenerative medicine

New CRISPR-Cas9 variant has potential to increase precision during gene therapy

New trial shows improved survival for patients with advanced colorectal BRAF-mutant cancer

Johns Hopkins researchers develop new way to deliver sight-saving gene therapy to the retina

New NIH program aims to improve lives for people with inherited blood and immune diseases

Study shows how AF4-MALS-UV can separate and size large AAV aggregates

7-year-old treated with FDA-approved gene therapy for Leber congenital amaurosis

Non-viral gene delivery method more effective and safer than standard viral approaches

Scientists to present new findings on gene therapy at Neuroscience 2019

Faster, cheaper, better: improved gene therapy tool production

Novel gene therapy for glycogen storage disease shows positive results

BUSM researchers develop advanced protocol to improve gene therapy tool production

The resurgence of gene therapy pioneer

Zylka lab receives NIH grant to advance treatments for neurodevelopmental disorders

Patients with non-Hodgkin's lymphoma may benefit from radiation, experience reduced side effects

Children's Tumor Foundation harnesses interest in rare disease research through NF Innovation Week

Nano-sized bubbles in our body could effectively fight cancer

New gene therapy helps stroke patients develop new neurons

New approach enables the body to produce specific antibodies

BIA Separations launches Cornerstone AAV Process Development Service to improve gene therapy production

Event guide: 9th Gene Therapy Development Summit 2026 eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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