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.

Proof-of-concept study holds promise for safer gene therapy techniques for treating immunodeficiencies

Researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications.

1 Nov 2023

Sickle cell disease patients willing to accept high risks for gene therapy cure

Individuals living with severe sickle cell disease (SCD) are highly interested in new, potentially curative gene therapy treatments and are willing to accept associated risks for a chance at a cure, according to a study published today in Blood Advances.

31 Oct 2023

Novel replacement strategy holds promise for the treatment of various genetic disorders

Severe Combined Immunodeficiencies (SCIDs) are a group of debilitating primary immunodeficiency disorders, primarily caused by genetic mutations that disrupt T-cell development. SCID can also affect B-cell and natural killer cell function and counts.

27 Oct 2023

Gene therapy breakthrough: Scientists develop method to deliver larger genes to target cells

Gene therapy currently represents the most promising approach for the treatment of hereditary diseases. Yet despite significant breakthroughs in recent years, there are still a number of hurdles that hinder the wider application of gene therapies.

24 Oct 2023

Scientists develop gene therapy approach for rare paralyzing disease

The 50 families stretch from the Netherlands and the United Kingdom to the United States and China. Each family has a child who is paralyzed from a mutation in a single gene named Contactin-Associated Protein 1 (Cntnap1).

20 Oct 2023

Research reveals new way viruses suppress the CRISPR-Cas immune systems of bacteria

A microscopic discovery will not only enable scientists to understand the microbial world around us but could also provide a new way to control CRISPR-Cas biotechnologies.

18 Oct 2023

FAST joins with University of Pennsylvania to develop investigational AAV gene therapy for Angelman syndrome

The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS).

17 Oct 2023

OrganaBio launches new division to meet growing demand for decentralized clinical trial support

OrganaBio, the Contract Technology Development and Manufacturing Organization (CTDMO) specializing in solutions for cell and immunotherapy development, today announces the official launch of Cell Processing and Cryopreservation (CPC) Services, a new division of OrganaBio.

12 Oct 2023

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

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

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.