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 drug molecule restores lost nerve-muscle connections, enhances strength

A small molecule previously shown to enhance strength in injured or old laboratory mice does so by restoring lost connections between nerves and muscle fibers, Stanford Medicine researchers have found.

11 Oct 2023

Recent Advancements in Cell Based Therapy

In this interview, NewsMedical talks to James Brady about cell-based therapy and how the field of genetic engineering transformed.

From MaxCyte, Inc. 11 Oct 2023

CRISPR gene editing study provides insights into how autism gene affects brain development

Researchers have used CRISPR gene editing, stem cells and human neurons to study the impact of a gene that is commonly mutated in autism.

5 Oct 2023

USC receives $2 million grant to advance cell and gene therapy research

The Keck School of Medicine of USC has received $2 million from the California Institute of Regenerative Medicine (CIRM) to further augment its newly launched cGMP Laboratory, a state-of-the-art facility designed to advance early-stage research into clinically viable cell and gene therapies.

4 Oct 2023

NIH grant to fund development of new gene therapy platform for brain diseases

A two-phase NIH grant will fund research into a new CRISPR-based gene therapy platform that will target genetic brain diseases like Angelman syndrome and H1-4 (HIST1H1E) syndrome.

3 Oct 2023

New CRISPR-based gene-editing tool could lead to better treatments for genetic disorders

A new CRISPR-based gene-editing tool has been developed which could lead to better treatments for patients with genetic disorders.

29 Sep 2023

Cutting-edge NanoSIMS imaging sheds light on the progression of Parkinson's disease

Parkinson's disease is a complex neurodegenerative disorder that leads to the deterioration of specific types of neurons in the brain, resulting in a number of motor and non-motor symptoms.

29 Sep 2023

Advancement of potential gene therapy could offer new hope for children with Batten disease

A new study shows that delivery of gene therapy to correct the gene mutations that cause CLN2 disease, or Batten disease, directly into the cerebrospinal fluid (CSF) has potential therapeutic effects.

28 Sep 2023

Rice researchers achieve noninvasive gene delivery to multiple brain regions

Rice University researchers tested the safety and feasibility of gene delivery to multiple brain regions using a noninvasive, ultrasound-based technique in rodents, and their findings suggest that the efficiency of gene delivery improves within each targeted site when more sites are opened.

27 Sep 2023

Multipronged gene therapy restores walking in mice after complete spinal cord injury

When the spinal cords of mice and humans are partially damaged, the initial paralysis is followed by the extensive, spontaneous recovery of motor function.

21 Sep 2023

Scientists uncover a crucial component for restoring functional activity after spinal cord injury

In a new study in mice, a team of researchers from UCLA, the Swiss Federal Institute of Technology, and Harvard University have uncovered a crucial component for restoring functional activity after spinal cord injury.

21 Sep 2023

Ongoing gene therapy trials have driven increased use of ALS genetic testing, finds study

Researchers at The Ohio State University Wexner Medical Center and College of Medicine led the creation of evidence-based consensus guidelines for genetic testing and counseling for patients with amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

19 Sep 2023

New research offers insight about the reason mitochondrial DNA is maternally inherited

New research provides insight about the bedrock scientific principle that mitochondrial DNA - the distinct genetic code embedded in the organelle that serves as the powerplant of every cell in the body - is exclusively passed down by the mother.

19 Sep 2023

Researchers identify key mechanisms to address future treatments for hereditary blindness

There are still many enigmas about the mechanism of action of the CERKL gene, which causes retinitis pigmentosa and other hereditary vision diseases.

18 Sep 2023

Broken String Biosciences closes $15M series A funding round

Broken String Biosciences today announced that it has closed a $15 million Series A investment round.

From Broken String Biosciences 18 Sep 2023

AACR Cancer Progress Report provides latest statistics on cancer research and treatment

Today, the American Association for Cancer Research (AACR) released the 13th edition of its annual Cancer Progress Report, which chronicles how basic, translational, and clinical cancer research and cancer-related population sciences - primarily supported by federal investments in the National Institutes of Health (NIH) and the National Cancer Institute (NCI) - remain vitally important to improving health and saving lives.

13 Sep 2023

Dormant HIV reservoirs can spontaneously produce RNA and proteins, study finds

HIV anti-retroviral therapy is considered a treatment and not a cure because patients usually carry a reservoir of HIV-infected cells that can re-emerge if treatment stops.

13 Sep 2023

Is DNA methylation the missing link in diagnosing and treating psoriasis and psoriatic arthritis?

Researchers across Europe have delved into the DNA methylation patterns in CD4+ T-cells of psoriasis and psoriatic arthritis (PsA) patients, unveiling potential new biomarkers for these conditions. Their study suggests that these unique profiles could serve as both diagnostic and prognostic tools, although further validation is needed for real-world clinical application.

10 Sep 2023

Ohio State University Wexner Medical Center administers first targeted gene therapy for ALS

The Ohio State University Wexner Medical Center is one of the first nationwide to administer a targeted gene therapy for patients with a specific form of amyotrophic lateral sclerosis (ALS), a neurodegenerative disease that affects the cells in the brain and spine.

8 Sep 2023

Newly discovered nanobody may lead to a treatment for Retinitis Pigmentosa

A team of scientists from the University of California, Irvine, believe they have discovered a special antibody which may lead to a treatment for Retinitis Pigmentosa, a condition that causes loss of central vision, as well as night and color vision.

7 Sep 2023