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...

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...

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...

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,...

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...

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...

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...

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 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...

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...

Sensorion Receives French Regulatory Approval to Initiate HearConnex Gene Therapy Trial for GJB2-Related Hearing Loss

Sensorion has received French regulatory approval to begin a Phase I/II trial of SENS-601, its investigational gene therapy for GJB2-related hearing loss, with first patient dosing...

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...

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.

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.

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

Teen's life transformed by gene editing

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

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...

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...

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...

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...

‘Smart’ Nanoparticles Deliver mRNA Directly to Tumors in New Cancer Therapy

The treatment can reprogram immune cells that have been corrupted by the cancer, strengthening the body’s ability to attack.

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...

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...

RNAV8 Bio Joins ARPA-H Team to Pioneer Programmable RNA Medicines

The PROPEL program aims to control gene expression through RNA structure, building logic-gated RNA therapies that switch on only where and when intended, without altering the genom...