Social Media Ideas for Gene Therapy

Scientists working toward next-generation hemophilia B gene therapy

A gene therapy that delivers instructions to produce a longer-lasting version of the clotting protein missing or deficient in hemophilia B may improve treatment effectiveness while...

A New RNA Strategy Could Target Thousands of Genetic Disorders

This new approach targets disorders including cystic fibrosis, muscular dystrophy, and some cancers. In lab-grown tissue from a cystic fibrosis patient who did not respond to exist...

Targeted Lentiviral Delivery Without Vector Reengineering

Rather than requiring genetic modification of the viral glycoprotein, G-Link uses a modular protein adaptor to blind native VSV-G tropism and redirect delivery after vector product...

Gene Therapy Extends Lifespan and Reduces Symptoms in Sandhoff Disease Cat Model

Intravenous gene therapy extended lifespan, reduced neurological symptoms, and improved disease markers in a Sandhoff disease cat model, supporting further investigation of systemi...

Creation of Chimeric Antigen Receptor Natural Killer Cells in Situ via Messenger RNA Therapy

Messenger RNA (mRNA) can be delivered into tissues and cells by encapsulating it into lipid nanoparticles (LNPs), and then injecting the nanoparticles. A target cell engulfs a LNP,...

Gene Therapy Shows Proof of Concept for Treating Hydrocephalus at Its Source

A University of Cincinnati and UC Davis team showed that an AAV gene therapy targeting cerebrospinal fluid-producing cells reduced hydrocephalus-related brain swelling in animal mo...

Germany treats 1st patient with CRISPR therapy since approval

A patient in Germany has been successfully treated with a drug based on CRISPR gene-editing technology for the first time since the therapy was approved, the Charité medical school...

Inhaled Suppressor tRNA Restores Gene Function in Cystic Fibrosis Models

A preclinical study showed how chemically enhanced suppressor tRNAs combined with a lung-targeted delivery system can restore production of a critical protein in models of cystic f...

Blind Patients Recover Partial Vision in Gene Therapy Trial

The treatment delivers a light-sensitive algae protein to the retina. Paired with special goggles, it can restore limited vision and lasts for years. The post Blind Patients Recove...

FDA approves first gene therapy treatment for Sanfilippo syndrome type A

The Abigail Wexner Research Institute at Nationwide Children's Hospital celebrated the Food and Drug Administration's (FDA) approval of FAYUVI for Sanfilippo syndrome type A, follo...

Grip Strength as Biomarker of Aging in the Context of Presently Available Gene Therapies

A range of gene therapies are presently available in the medical tourism community of clinics outside the US and Europe, largely originating with a small number of companies such a...

Prime Assembly Expands Genome Editing with Precise, Large-Scale DNA Integration

A new genome-editing method, prime assembly, precisely inserts long DNA sequences into targeted genomic locations, potentially enabling mutation-agnostic gene therapies for diverse...

Rewriting the rules of genetic medicine with tRNA therapeutics

Our guest today is Dr. Nerissa Kreher, Chief Medical Officer of Alltrna. Nerissa brings two decades of rare disease drug development experience. The post Rewriting the rules of gen...

Wiskott-Aldrich syndrome: Long-term results of gene therapy developed

A single infusion of genetically corrected autologous hematopoietic stem cells can provide lasting clinical benefits for patients with Wiskott-Aldrich syndrome, a rare genetic diso...

NewBiologix and Synastra Join Forces to Advance rAAV Manufacturing for Duchenne Gene Therapy

The collaboration brings together Synastra’s expertise in genomic engineering, AAV vector design, rare-disease gene therapy, and translational development with NewBiologix’s propri...

Gene therapy helps doctors restore hearing for children born deaf

A major medical triumph is now giving some children born deaf the ability to hear.

Lipid Nanoparticles Deliver CAR Instructions to T Cells Without Viruses

Researchers have engineered a custom ionizable lipid nanoparticle that delivers CAR-encoding mRNA into human T cells with over 90 percent efficiency, producing potent antitumor act...

Gene therapy helps doctors restore hearing for children born deaf

A major medical triumph is now giving some children born deaf the ability to hear.

Andelyn Curator Biomanufacturing Platform Supports Fayuvi Gene Therapy Approval

Sanfilippo syndrome type A (MPS IIIA) is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginnin...

Teen's life transformed by gene editing

Vian, 16, has thalassemia and had to have monthly blood transfusions throughout her childhood.

CAR T-Cell Therapy Puts Severe Rheumatoid Arthritis Into Remission in First Clinical Trial

Researchers at Charité report promising results from the first CAR T-cell trial for treatment-refractory rheumatoid arthritis, with all six patients improving and three achieving s...

Targeted Reduction of PTBP1 Reverses Cognitive Impairment in a Mouse Model of Alzheimer's Disease

Many of the more sophisticated forms of medicine, including gene therapies and immunotherapies, are challenging to deliver to the brain. At the very least there are far fewer pract...

Frost & Sullivan presents the “Global Gene Therapy Crdmo Technology Innovation Leadership Award” to PackGene Biotech...

Andelyn to Support Manufacturing Scaleup for Genprex’s Diabetes Gene Therapy

Under the agreement, Andelyn will transition Genprex's adeno-associated virus (AAV)-based diabetes gene therapy candidate with process optimization, scale-up, and IND-enabling and...