Gene Therapy News

July 26, 2024

Top Headlines

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 muscular dystrophy ...

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 the future, repairing those muscles. The research ...

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 the animals to regain hearing. The approach may ...

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 ...

Latest Headlines

updated 8:52pm EDT

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 ...

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 ...

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 ...

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 ...

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 ...

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 ...

Genetic Signature May Predict Response to Immunotherapy for Non-Small Cell Lung Cancer

Feb. 23, 2024 — A new study identified a set of 140 genes that may help predict enhanced disease-free survival in patients with non-small cell lung cancer (NSCLC) treated with a combination of immunotherapy and ...

Gene-Editing Offers Hope for People With Hereditary Disorder

Feb. 2, 2024 — A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead ...

Researchers Identify Key Characteristics Associated With Improved CAR T Outcomes in Large B Cell Lymphoma

Jan. 17, 2024 — Axi-cel CAR T targets the CD19 molecule on large B-cell lymphoma cells. The ZUMA-7 trial demonstrated that axi-cel reduced the risk of disease progression, the need for new therapy, or death by 60% ...

A Novel Switch to Turn Genes On/off on Cue, a Promising Step Toward Safer Gene Therapy

Jan. 2, 2024 — Just like a doctor adjusts the dose of a medication to the patient's needs, the expression of therapeutic genes, those modified in a person to treat or cure a disease via gene therapy, also ...

Mice Possess Natural Gene Therapy System

Dec. 13, 2023 — A previously mysterious small RNA molecule in mice is found to play a crucial role in gene expression, and may be the first identified member of a new class of regulatory ...

Promising New Gene Therapy Approach for Genetic Heart Disease: Clinical Trials Imminent

Dec. 7, 2023 — Researchers have laid the foundation for the development of a gene therapy for the genetic heart disease arrhythmogenic cardiomyopathy (ACM). Their approach, based on replacement of the PKP2 gene, ...

Pediatric Oncology: Scientists Discover New Achilles Heel of Leukemia Cells

Nov. 22, 2023 — Leukemia is the most common type of cancer in children. Treatment involves intensive chemotherapy, which has severe side effects due to its non-specific mode of action. A team has now discovered a ...

New Research Advances Understanding of Cancer Risk in Gene Therapies

Nov. 16, 2023 — Researchers have discovered that 'cell competition' following gene therapy results in the accumulation of stem cells with genetic mutations which make them grow faster. Some of these ...

New Nijmegen Method Reveals Hidden Genetic Variations

Nov. 1, 2023 — Many hidden genetic variations can be detected with Chameleolyser, a new method. The information is already yielding new patient diagnoses and may also lead to the discovery of as yet unknown disease ...

Individuals With Severe Sickle Cell Disease Express High Risk Tolerance for Gene Therapies

Oct. 31, 2023 — 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, ...

New Methods for Effective Transport of Large Genes in Gene Therapy

Oct. 24, 2023 — One problem in gene therapy is that not all genes transfer equally well into the target cells. Researchers have now developed a flexible method to transfer large genes efficiently and without ...

Promising Evidence for Sickle Cell Gene Therapy

Aug. 30, 2023 — A clinical trial has tested a potentially curative stem cell gene therapy for sickle cell disease. The results were ...

Research Raises Hopes for New Treatment of Fusion-Driven Cancer

Aug. 11, 2023 — Researchers have developed a new form of gene therapy that can stop cell division in fusion-driven ...

New Gene Editing Tool Helps Zero in on Small Cancer-Linked Mutations

Aug. 10, 2023 — A change in just one letter in the code that makes up a cancer-causing gene can significantly affect how aggressive a tumor is or how well a patient with cancer responds to a particular therapy. A ...

Gene Therapy Hope for Children With Kidney Disease

Aug. 10, 2023 — Researchers have made a remarkable step forward in finding a potential cure for a type of childhood kidney ...