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.

Virally Mediated Gene Therapy Rescues Hearing in Aging Mice

By 2050, one in 10 individuals are expected to live with some form of hearing loss. Of the hundreds of millions of cases of hearing loss affecting individuals worldwide, genetic hearing loss is often the most difficult to treat.

29 May 2023

Stimulating Cell DNA Repair Processes to Combat the Root Cause of Autism Spectrum Disorders

A new treatment option has been discovered for fragile X syndrome, which is the most common genetic cause of autism spectrum disorders and is defined by a hereditary repetition of certain nucleotides in the DNA sequence of the FMR1 gene. Researchers at Massachusetts General Hospital (MGH) carried out the study, which was published in the journal Cell.

22 May 2023

Study Uncovers Cancer-Like Changes in Mitochondrial Disease of Newborns

A study found that a mitochondrial disease of newborns shows cancer-like changes in proliferating cells, causing tissues to age prematurely.

4 May 2023

New Approach Could Power the Development of Advanced Cell Therapies

A new approach to the genetic engineering of cells promises significant improvements in speed, efficiency, and reduction in cellular toxicity compared to current methods.

2 May 2023

New Gene Therapy may be on the Horizon for Usher Syndrome Type 1F

Usher Syndrome type 1F is a rare but severe genetic disease that causes deafness, lack of balance, and progressive blindness.

27 Apr 2023

UH Researcher Designs New Model Expressing the Most Common Disease Mutation in USH2A

Usher syndrome, a rare inherited genetic disease, is a leading cause of combined deafness and blindness with type 2A (USH2A) being the most common form.

18 Apr 2023

Optimized Genome-Editing Method Vastly Reduces Mutations

CRISPR-Cas9 is frequently used to alter the genome by researching and editing disease-related genes. However, this approach is coupled with undesirable side effects including mutations and toxicity.

11 Apr 2023

Study Paves the Way to Targeted Treatments for Stargardt Disease

A recent study in the Journal of Biological Chemistry revealed the key to a protein that commonly causes blindness.

5 Apr 2023

Newly developed toolkit uses small molecules to control the activity of synthetic RNA

The Okinawa Institute of Science and Technology, in collaboration with Astellas Pharma Inc., has developed a new toolkit that uses small molecules to control the activity of a piece of synthetic RNA, and ultimately regulate gene expression.

31 Mar 2023

Single monoclonal antibody infusion can strengthen the body's fight against yellow fever

New research from Oregon Health & Science University and collaborators indicates lab-made antibodies may be able to cure people infected with yellow fever, a virus for which there is no treatment.

30 Mar 2023

FDA-approved compound promotes photoreceptor survival in lab models of inherited retinal ciliopathy

A National Eye Institute-led team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 (LCA 10), an inherited retinal ciliopathy disease that often results in severe visual impairment or blindness in early childhood.

30 Mar 2023

Study suggests new strategy for treating arrhythmogenic cardiomyopathy

University of Utah Health scientists have corrected abnormal heart rhythms in mice by restoring healthy levels of a protein that heart cells need to establish connections with one another.

20 Mar 2023

Using a programmable control switch to deliver therapeutic RNA molecules to target cells

Using an RNA sensor, MIT engineers have designed a new way to trigger cells to turn on a synthetic gene.

17 Mar 2023

Age-related sarcopenia prevented by AAV1.NT-3 gene therapy

A new research paper was published on the cover of Aging (listed by MEDLINE/PubMed as "Aging (Albany NY)" and "Aging-US" by Web of Science) Volume 15, Issue 5, entitled, "AAV1.NT-3 gene therapy prevents age-related sarcopenia."

16 Mar 2023

Scientists discover two ways to preserve diseased upper motor neurons in ALS

Northwestern Medicine scientists have discovered two ways to preserve diseased upper motor neurons that would normally be destroyed in ALS, based on a study in mice. Upper motor neurons initiate movement, and they degenerate in ALS.

24 Feb 2023

MD Anderson and Replay launch new company pioneering T cell receptor NK cell therapies

The University of Texas MD Anderson Cancer Center and Replay today announced the launch of Syena, a new oncology-focused product company pioneering T cell receptor (TCR) natural killer (NK) cell therapies.

15 Feb 2023

UChicago scientists shed light on the longstanding puzzle of RNA methylation

University of Chicago scientists have discovered a new wrinkle in our understanding of how our genes work.

14 Feb 2023

Cerba Research and Teddy Lab sign Memorandum of Understanding on entering into a discussion for Joint Venture

Cerba Research announced today a Memorandum of Understanding with Teddy Clinical Research Laboratory (Wuxi, China), a preeminent central lab provider covering mainland China, to lay the foundation for an in-depth discussion towards the creation of a Joint Venture.

From Cerba Research 3 Feb 2023

CHOP scientists build a custom tool that drastically reduces engraftment reporting error rates

Researchers at Children's Hospital of Philadelphiahave developed a custom-built application to automate determination of engraftment, a key outcome after hematopoietic stem cell transplant (HSCT).

31 Jan 2023

Innovative gene therapy to cure multiple eye diseases

The future treatment of individuals with various eye diseases could benefit immensely from a potential new gene therapy strategy, according to Trinity University researchers.