Gene Therapy News

June 13, 2025

Top Headlines

Experimental Drug May Benefit Some Patients With Rare Form of ALS

May 23, 2025 — Some patients with a rare form of ALS benefited from an experimental therapy, with biomarker evidence of reduced injury to neurons and even limited functional ...

Researchers Develop Gene Therapy That Can Target Airway and Lungs Via Nasal Spray

May 22, 2025 — For gene therapy to work well, therapeutic molecules need to be efficiently delivered to the correct locations in the body -- a job commonly given to adeno-associated viruses (AAV). To improve the ...

New Gene Editor Enables Greater Precision

May 15, 2025 — A new gene editor may soon open the door to gene therapies for a wider array of ...

Infant With Rare, Incurable Disease Is First to Successfully Receive Personalized Gene Therapy Treatment

May 15, 2025 — A research team has developed and safely delivered a personalized gene editing therapy to treat an infant with a life-threatening, incurable genetic disease. The infant, who was diagnosed with the ...

Duchenne Muscular Dystrophy Gene Therapy

May 14, 2025 — Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene ...

Gene Circuits Enable More Precise Control of Gene Therapy

Apr. 28, 2025 — To help achieve more precise control of gene therapy, engineers have designed a new control circuit that can keep gene expression levels within a target range. The method could be used to deliver genes that could help treat diseases including ...

Setback in Gene Therapy for Duchenne Muscular Dystrophy as Immune System Emerges as Key Barrier

Apr. 8, 2025 — A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective ...

New Gene Editing Tool Shows Promise for Treating Diseases With Multiple Mutations

Apr. 9, 2025 — Investigators have developed STITCHR, a new gene editing tool that can insert therapeutic genes into specific locations without causing unwanted mutations. The system can be formulated completely as ...

One Gene Variant Is Poised to Cure a Devastating Inherited Disease

Feb. 27, 2025 — For more than 15 years, a pulmonologist has tried to understand the random path of devastation that a rare genetic condition carves through the families it ...

Gene Therapy for Rare Epilepsy Shows Promise in Mice

Feb. 14, 2025 — Recent research takes aim at the a variant in gene SCN1B, which causes a severe form of developmental epileptic ...

Gene Modification Could Improve Cancer Treatment's Success Rate

Feb. 7, 2025 — A research team has made a major discovery on how the CUL5 gene affects CAR-T cell therapy, an advanced cancer therapy. The study reveals that reducing the activity of the CUL5 gene helps CAR-T cells fight cancer more effectively. These findings ...

Light-Induced Gene Therapy Disables Cancer Cells' Energy Center

Dec. 13, 2024 — Researchers are shining a light on cancer cells' energy centers -- literally -- to damage these power sources and trigger widespread cancer cell death. In a new study, scientists combined strategies to deliver energy-disrupting gene therapy using ...

Latest Headlines

updated 12:03pm EDT

New Gene Therapy Reverses Heart Failure in Large Animal Model

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the amount of blood the ...

Researchers Discover Underlying Mechanisms That Make CRISPR an Effective Gene Editing Tool

Oct. 28, 2024 — Using state-of-the-art technology, researchers have identified several specific steps needed for CRISPR to become active and perform its gene editing ...

Gene Therapy Shows Long-Term Benefit for Patients With a Rare Pediatric Brain Disease

Oct. 9, 2024 — Cerebral adrenoleukodystrophy (CALD) is a rare progressive, genetic brain disease that primarily presents in young boys, causing loss of neurological ...

Single-Dose Gene Therapy Is Potentially Life-Changing for Adults With Hemophilia B

Sep. 25, 2024 — Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to ...

New Gene Therapy Offers Promise for Treating Glaucoma -- As Well as AMD

Sep. 9, 2024 — Scientists developed a highly promising gene therapy to treat glaucoma -- a debilitating eye condition that can lead to complete vision loss, and which affects around 80 million people worldwide. The ...

Effective New Gene Therapy to Treat Multiple Sulfatase Deficiency

Sep. 9, 2024 — Researchers tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, skin, and skeleton with no ...

Gene Therapy Gets a Turbo Boost

Aug. 29, 2024 — For decades, scientists have dreamt of a future where genetic diseases, such as the blood clotting disorder hemophilia, could be a thing of the past. Gene therapy, the idea of fixing faulty genes ...

Noninvasive Measurement of Gene Expression at Target Locations in the Brain

Aug. 7, 2024 — Bioengineers have developed a noninvasive tool to measure gene expression and gene therapy delivery in specific brain regions using ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy

July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne ...

New Gene Therapy for Muscular Dystrophy Offers Hope

July 17, 2024 — A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic disease, but perhaps, in ...

With Gene Editing, Mice With a Form of Inherited Deafness Can Hear Again

July 12, 2024 — Researchers have used gene editing to restore hearing in adult mice with a type of inherited hearing loss. They showed that shutting down a damaged copy of a gene called a microRNA (miRNA) enabled ...

Novel Genome Editing Approach Restores Hearing in Adult Preclinical Models With Genetic Deafness

July 10, 2024 — Researchers restored hearing in preclinical mouse models with a specific form of inherited deafness called DFNA50 caused by mutations in microRNA, by using a novel in vivo CRISPR genome editing ...

Researchers Discover Potential Mole Reversal Therapy in Rare Condition

June 17, 2024 — Researchers have designed a new genetic therapy that could alleviate debilitating giant moles in a rare skin ...

Promising Gene Therapy for FOXG1 Syndrome

June 13, 2024 — A viral gene therapy has reversed some brain abnormalities in infant mice with FOXG1 syndrome, a significant step toward one day treating children with this severe neurodevelopmental ...

Improved Prime Editing System Makes Gene-Sized Edits in Human Cells at Therapeutic Levels

June 10, 2024 — Scientists have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for ...

New Gene Therapy Trial Shows Restored Hearing and Speech in Children Born Deaf, Treated in Both Ears

June 5, 2024 — A new clinical trial of five children with inherited deafness found administering gene therapy in both ears led to restored hearing and speech, and additional gains including sound source ...

New, Modified CRISPR Protein Can Fit Inside Virus Used for Gene Therapy

May 30, 2024 — Researchers have developed a novel version of a key CRISPR gene-editing protein that shows efficient editing activity and is small enough to be packaged within a non-pathogenic virus that can deliver ...

Could a Medicated Foam Make Gene Therapies More Accessible?

May 29, 2024 — Bioengineers report that a foaming liquid worked better than a standard liquid formulation at transferring gene therapy components to cells in laboratory ...

New Gene Delivery Vehicle Shows Promise for Human Brain Gene Therapy

May 16, 2024 — In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain ...

An Electrifying Discovery May Help Doctors Deliver More Effective Gene Therapies

Apr. 30, 2024 — In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering has developed a stimulating method that could make the human body more receptive to ...

Gene-Based Therapy Restores Cellular Development and Function in Brain Cells from People With Timothy Syndrome

Apr. 24, 2024 — In a proof-of-concept study, researchers demonstrated the effectiveness of a potential new therapy for Timothy syndrome, an often life-threatening and rare genetic disorder that affects a wide range ...

Mosaics of Predisposition Cause Skin Disease

Apr. 22, 2024 — Clarifying the cause of a skin disease led to the discovery of a new disease-causing gene, a new category of diseases, and new perspectives for both counseling and therapy. The discovery is the first ...

Nanoparticle Delivery of FZD4 to Lung Endothelial Cells Inhibits Lung Cancer Progression and Metastases

Apr. 16, 2024 — A recent breakthrough study has shown potential to improve therapeutic outcomes for patients suffering from lung ...

New Insight Into Combating Drug-Resistant Prostate Cancer

Apr. 10, 2024 — New research sheds light on the significance of the glucocorticoid receptor in drug-resistant prostate cancer, showing that the development of drug resistance could be prevented by limiting the ...

An Hereditary Liver Disease Cured With the Help of Gene Scissors

Apr. 4, 2024 — Researchers have succeeded in correcting a gene defect that causes a hereditary liver disease and its adverse effects on ...

Genetically Engineered Dendritic Cells Enhance the Power of Immunotherapy Against Lung Cancer

Mar. 26, 2024 — A new study suggest that using CXCL9 and CXCL10-producing dendritic cells alongside immunotherapy can be a promising strategy to overcome treatment resistance and improve clinical outcomes for ...

An Immunotherapy to Overcome Resistant Leukemia

Mar. 21, 2024 — Researchers have discovered that a specific mutation in the cancer cells of an aggressive type of blood cancer can prevent novel immunotherapies such as CAR T-cell therapy from working. Their study ...

Gene Discovered That Can Protect Against Severe Muscle Disease

Mar. 6, 2024 — A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, muscular ...

Researchers Closing in on Genetic Treatments for Hereditary Lung Disease, Vision Loss

Mar. 5, 2024 — Researchers who work with tiny drug carriers known as lipid nanoparticles have developed a new type of material capable of reaching the lungs and the eyes, an important step toward genetic therapy ...

A Better Way to Deliver Fetal Therapy for Serious Genetic Disorders

Mar. 4, 2024 — In a discovery that opens the door to a less invasive way of treating some serious disorders before birth, UC San Francisco scientists have found that delivering medicine through amniotic fluid is as ...