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.

Pioneering gene therapy has long-lasting effects on children with deadly immune disorder

Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.

18 Oct 2021

Researchers identify the mechanism leading to deafness in Norrie disease

A team of Mass Eye and Ear Scientists led by Albert Edge, PhD, working with research fellow Yushi Hayashi, MD, PhD, has identified the mechanism that can lead to deafness in the rare syndrome, Norrie disease.

6 Oct 2021

Gene therapy turns glial brain cells into neurons, restores visual function

A team of scientists identified a means to employ gene therapy to convert glial brain cells into neurons, reinstating visual function.

4 Oct 2021

Supercoiling and looping can transmit mechanical stress along the DNA backbone, shows study

When people think of DNA, they visualize a string-like double helix structure. In reality, the DNA double helix in cells is supercoiled and constrained into loops. This supercoiling and looping are known to influence every aspect of DNA activity, but how this happens has not been clear.

28 Sep 2021

Exploring cell and gene based therapies for Parkinson’s disease

After several decades of pre-clinical development, cell- and gene-based therapies for Parkinson's disease (PD) are now actively being explored. In this special supplement to the Journal of Parkinson's Disease on "Repairing the Parkinsonian Brain," experts highlight some of the current strategies being pursued to restore lost function and replace what is lost in the PD brain, with special emphasis on the challenges associated with translating advanced therapeutic approaches into pioneering clinical trials.

24 Sep 2021

Researchers develop novel cell reprogramming strategy to slow down glioblastomas

Gliomas are the most common primary brain tumors in adults. Among them, high-grade glioblastomas (GBMs) are particularly known to be notoriously aggressive and invasive, which makes it challenging to treat them.

16 Sep 2021

Study explores the efficiency of CasMINI, an engineered CRISPR-Cas system

A compact CRISPR-Cas system, known as CasMINI, can be employed for gene-therapy and cell-engineering applications as it is easier to deliver into cells.

7 Sep 2021

Miniature CRISPR-Cas system expands applicability and implications further

For the first time, Stanford researchers have miniaturized CRISPR for mammalian gene editing, which magnifies the scope of CRISPR technology.

3 Sep 2021

New study could help develop novel treatments for neurodegenerative diseases

Researchers have introduced a novel gene therapy program that could set the stage for new treatments for neurodegenerative diseases.

2 Aug 2021

Modification in some RNA molecules could protect against non-alcoholic fatty liver

RNA molecules while carrying genetic instructions from DNA to the protein-making machinery of cells could help guard against non-alcoholic fatty liver.

21 Jul 2021

Gene therapy may be safe and effective in treating AADC deficiency

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency.

19 Jul 2021

Novel gene therapy offers hope to children with genetic, neurodegenerative diseases

Newly devised gene therapy can help children born with AADC, a rare genetic disorder that causes developmental and physical disabilities.

13 Jul 2021

Children with rare genetic disorder experience dramatic improvements in a gene therapy trial

Children with a devastating genetic disorder characterized by severe motor disability and developmental delay have experienced sometimes dramatic improvements in a gene therapy trial launched at UCSF Benioff Children's Hospitals.

13 Jul 2021

Novel platform may lead to more improved gene therapies

Researchers from RCSI University of Medicine and Health Sciences have developed polypeptide-based materials that serve as effective vectors in delivering gene therapies.

11 Jun 2021

Gene therapy may be a safe and effective single-dose treatment for hypophosphatasia

A preclinical study led by scientists at Sanford Burnham Prebys has established that AAV8-TNAP-D10--a gene therapy that replaces a key enzyme found in bone--may be a safe and effective single-dose treatment for hypophosphatasia (HPP).

8 Jun 2021

New software tool detects, evaluates, and quantifies off-target editing activity

CRISPR technology enables researchers to edit genomes by modifying DNA sequences and hence gene function.

27 May 2021

New study assesses total economic impact of human genomics

Human genetics and genomifefcs contributed $265 billion to the U.S. economy in 2019 and has the potential to drive significant further growth given major new areas of application, according to a new report issued today by the American Society of Human Genetics (ASHG).

19 May 2021

Lack of functional GAS2 gene causes severe hearing impairment in mice

A gene called GAS2 plays a key role in normal hearing, and its absence causes severe hearing loss, according to a study led by researchers in the Perelman School of Medicine at the University of Pennsylvania.

10 May 2021

New gene therapy could help treat Duchenne muscular dystrophy

Researchers from UT Southwestern used a new kind of gene therapy to successfully treat mice with Duchenne muscular dystrophy (DMD), distinctively employing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein missing in several DMD patients.

3 May 2021

National DNA Day 2021: An Interview with Professor George Church

In celebration of National DNA Day, AZoLifeSciences interviews renowned DNA expert Professor George Church about his life-long career in DNA research.

From Harvard Medical School 23 Apr 2021