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.

New microcapsules represent promising vectors for application of genome-editing tools

Researchers from Tomsk Polytechnic University jointly with their colleagues from St. Petersburg, Hamburg and London have conducted a study in the course of which it was found out that polymer and hybrid silica-coated microcapsules are more efficient in genome-editing when applying CRISPR-Cas9 system.

6 Oct 2017

CGT Catapult’s iPSC line licensed to Universal Cells for creating universal donor cell line

The Cell and Gene Therapy Catapult today announced a non-exclusive licensing agreement to provide its clinical-grade induced Pluripotent Stem Cell line to Seattle based Universal Cells.

6 Oct 2017

Moms of children with rare genetic illness push for wider newborn screening

Kerri De Nies received the news this spring from her son's pediatrician: Her chubby-cheeked toddler had a rare brain disorder.

5 Oct 2017

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

In a recent clinical trial, a gene therapy to treat cerebral adrenoleukodystrophy (CALD) -- a neurodegenerative disease that typically claims young boys' lives within 10 years of diagnosis -- effectively stabilized the disease's progression in 88 percent of patients, researchers from the Dana-Farber/Boston Children's Cancer and Blood Disorders Center and Massachusetts General Hospital report today.

4 Oct 2017

New gene therapy to treat cancer in children, young adults offers hopes and challenges

The first gene therapy for cancer, approved by the Food Drug Administration in August, will transform the treatment of a particular kind of cancer in children and young adults. It's transformative because it uses a patient's own immune cells to attack the cancer cells.

3 Oct 2017

Novel approach facilitates long-term persistence of AAV vector genomes in proliferating cells

Researchers added a scaffold/matrix attachment region (S/MAR) to a conventional adeno-associated virus (AAV) vector used for gene transfer, and the modified vectors were able to establish colonies and maintain long-term transgene expression in HeLa cells, as reported in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers.

22 Sep 2017

Researchers use gene therapy to restore hearing and balance in mouse model of Usher syndrome

Hearing loss, sometimes associated with other disorders such as balance defects, is the most common sensory deficit, affecting more than 280 million people worldwide, according to WHO. In France, one child in 700 is born with severe or profound hearing loss, and one in every 1,000 will lose their sense of hearing before adulthood.

22 Sep 2017

Novel gene therapy can inhibit, reverse multiple sclerosis in mouse models

Multiple sclerosis can be inhibited or reversed using a novel gene therapy technique that stops the disease's immune response in mouse models, University of Florida Health researchers have found.

21 Sep 2017

Gene therapy could be novel approach to treat alcoholism, research suggests

Researchers used gene transfer to block the expression of one of the two main enzymes that break down alcohol in the liver, leading to the accumulation in liver cells of acetaldehyde, a metabolic byproduct of ethanol.

19 Sep 2017

Scientists take important step toward gene therapy for deaf patients

Scientists at Oregon State University have taken an important step toward gene therapy for deaf patients by developing a way to better study a large protein essential for hearing and finding a truncated version of it.

19 Sep 2017

Research could lead to improvements in treating diseases caused by mutations in genes

Research carried out by academics at Northumbria University, Newcastle could lead to improvements in treating patients with diseases caused by mutations in genes, such as cancer, cystic fibrosis and potentially up to 6,000 other inherited conditions.

19 Sep 2017

MSC transplantation can be safe, effective approach to treat Crohn's disease-related fistula

A growing body of clinical evidence shows that transplantation of a patient's own mesenchymal stem cells (MSCs) to achieve a cure and prevent recurrent of Crohn's disease-related fistula can be a safe and effective addition to surgery.

14 Sep 2017

MGH researchers identify potential strategy for improving efficacy of angiogenesis inhibitors

A Massachusetts General Hospital research team has identified a potential strategy for improving the efficacy of angiogenesis inhibitors, drugs that help fight cancer by blocking the formation of new blood vessels.

13 Sep 2017

New testing regime could speed up diagnosis and treatment of SCID in infants

Babies born with Severe Combined Immune Deficiency (SCID) syndrome are defenceless against bacterial and viral infections that would be virtually harmless to most healthy people. If untreated, SCID is often fatal within a baby's first year of life.

13 Sep 2017

CGT Catapult partners with TrakCel to incorporate tracking technology into cell and gene therapy manufacturing centre

The Cell and Gene Therapy Catapult (CGT Catapult) today announces a partnership agreement with UK-headquartered company TrakCel to incorporate its needle-to-needle supply chain management platform into the CGT Catapult’s large-scale cell and gene therapy manufacturing centre.

8 Sep 2017

Gene Therapy News and Research

Further Reading

New microcapsules represent promising vectors for application of genome-editing tools

CGT Catapult’s iPSC line licensed to Universal Cells for creating universal donor cell line

Moms of children with rare genetic illness push for wider newborn screening

Gene therapy effectively stabilizes progression of cerebral adrenoleukodystrophy, trial reveals

New gene therapy to treat cancer in children, young adults offers hopes and challenges

Novel approach facilitates long-term persistence of AAV vector genomes in proliferating cells

Researchers use gene therapy to restore hearing and balance in mouse model of Usher syndrome

Novel gene therapy can inhibit, reverse multiple sclerosis in mouse models

Gene therapy could be novel approach to treat alcoholism, research suggests

Scientists take important step toward gene therapy for deaf patients

Research could lead to improvements in treating diseases caused by mutations in genes

MSC transplantation can be safe, effective approach to treat Crohn's disease-related fistula

MGH researchers identify potential strategy for improving efficacy of angiogenesis inhibitors

New testing regime could speed up diagnosis and treatment of SCID in infants

CGT Catapult partners with TrakCel to incorporate tracking technology into cell and gene therapy manufacturing centre

Penn researchers find abnormally shortened telomeres in young DMD patients’ muscle stem cells

UChicago Medicine working with Novartis to offer breakthrough gene therapy for pediatric ALL patients

FDA makes the first gene therapy Kymriah available for treatment of pediatric ALL

Pioneering cancer gene therapy gets green light — and $475,000 price tag

FDA approves first cell-based gene therapy for use in the United States

Ultimate guide to the essential tools for the characterization of proteins, polymers and nanoparticles 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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