Gene Therapy News

April 20, 2024

Top Headlines

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 activity of coregulator ...

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 also explains why the cancer cells are resistant ...

Latest Headlines

updated 10:46am EDT

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 ...

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) ...

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 ...

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 ...

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 ...

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 ...

Scientists Reverse Hearing Loss in Mice

Aug. 10, 2023 — New research has successfully reversed hearing loss in mice. Scientists used a genetic approach to fix deafness in mice, restoring their hearing abilities in low and middle frequency ...

Research Sheds New Light on Gene Therapy for Blood Disorders

Aug. 9, 2023 — A study shows new steps toward more patients getting gene ...

Researchers Develop 'in Vivo' RNA-Based Gene Editing Model for Blood Disorders

July 27, 2023 — In a step forward in the development of genetic medicines, researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the ...

Researchers Pinpoint Protein Tied to Drug Resistance in Patients With Lung Cancer

July 12, 2023 — Cancer therapies that target specific genetic abnormalities in tumors have revolutionized treatment possibilities over the past two decades. While quality of life and survival are improved with ...

Researchers Design an Innovative Strategy to Fight Obesity Through Gene Therapy

May 30, 2023 — A scientific team has designed a strategy to fight obesity and diabetes in mice through ex vivo gene therapy which consists of implanting cells that have been manipulated and transformed in order to ...

Peering Into Ocular Waste Recycling

Apr. 4, 2023 — Researchers uncover a mechanism in a retinal transporter protein that causes blindness, could lead to targeted ...

The Drug Fasudil Is Found to Reverse Key Symptoms of Schizophrenia in Mice

Feb. 17, 2023 — Researchers used the drug fasudil to restore neurons and improve methamphetamine-induced cognitive dysfunction in a mouse model of ...

Researchers Circumvent Radiation Resistance in Subtype of Brain Tumors

Jan. 24, 2023 — Researchers have uncovered a gene that is overexpressed in mutated IDH1. Studies in human cells and a novel mouse model both show that this gene, called ZMYND8, plays a critical role in the radiation ...

Researchers Take Key Step Toward New Treatment for Hereditary Blindness

Jan. 11, 2023 — Scientists have demonstrated in animal models the possibility of using lipid nanoparticles and messenger RNA, the technology underpinning COVID-19 vaccines, to treat blindness associated with a rare ...

Liver Cancer Study Encourages Caution With Certain Gene Therapies

Dec. 7, 2022 — Research has found that misfolded proteins in liver cells contribute to the development of liver cancer, shedding new light on the mysterious origins of one of the world's deadliest diseases. ...

Silicon Nanochip Could Treat Traumatic Muscle Loss

Nov. 9, 2022 — Technology that can change skin tissue into blood vessels and nerve cells also shows promise as a treatment for traumatic muscle ...

Gene Therapy Targeting Overactive Brain Cells Could Treat Neurological Disorders

Nov. 3, 2022 — A new treatment for neurological and psychiatric diseases, that works by reducing the excitability of overactive brain cells, has been ...