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.

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

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

Researchers from the Perelman School of Medicine at the University of Pennsylvania have made a discovery about muscular dystrophy disorders that suggest new possibilities for treatment.

7 Sep 2017

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

The University of Chicago Medicine is one of a limited number of U.S. sites working to offer a breakthrough gene therapy for pediatric acute lymphoblastic leukemia (ALL), which was just approved by the U.S. Food and Drug Administration.

31 Aug 2017

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

The U.S. Food and Drug Administration (FDA) has issued a press release making the first even gene therapy available for treatment of children with a form of acute lymphoblastic leukemia (ALL).

30 Aug 2017

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

The country's first approved gene therapy — approved Wednesday to fight leukemia that resists standard therapies — will cost $475,000 for a one-time treatment, its manufacturer announced.

30 Aug 2017

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

The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treatment of cancer and other serious and life-threatening diseases.

30 Aug 2017

Immunotherapy that uses existing cancer drugs in a new way delivers promising results

Immunotherapy is an emerging field in the global fight against cancer, even though scientists and clinicians have been working for decades to find ways to help the body's immune system detect and attack cancerous cells.

29 Aug 2017

Junk genes could one day offer framework for treating people with familial hypercholesterolemia

Scientists from UCLA and the Howard Hughes Medical Institute successfully used a gene that suppresses cholesterol levels as part of a treatment to reduce plaque in mice with a disorder called familial hypercholesterolemia.

26 Aug 2017

Researchers identify important adenovirus receptor in macrophages of mice

Adenoviral infections have a mild disease progression in healthy people, but it can be dangerous for immunocompromised people.

24 Aug 2017

Emory scientists use new tools to detect granulins for the first time inside cells

Emory University School of Medicine researchers have developed tools that enable them to detect small proteins called granulins for the first time inside cells.

14 Aug 2017

New version of gene editing technique corrects molecular mistakes in RNA-based diseases

Until recently, the CRISPR-Cas9 gene editing technique could only be used to manipulate DNA. In a 2016 study, University of California San Diego School of Medicine researchers repurposed the technique to track RNA in live cells in a method called RNA-targeting Cas9 (RCas9).

11 Aug 2017

Gene Therapy News and Research

Further Reading

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

Immunotherapy that uses existing cancer drugs in a new way delivers promising results

Junk genes could one day offer framework for treating people with familial hypercholesterolemia

Researchers identify important adenovirus receptor in macrophages of mice

Emory scientists use new tools to detect granulins for the first time inside cells

New version of gene editing technique corrects molecular mistakes in RNA-based diseases

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