Gene Therapy News

December 1, 2023

Top Headlines

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 site in the DNA of cancer cells that is essential ...

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 genetic mutations have been previously seen at higher levels in ...

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 ...

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 significant side effects. The approach has strong ...

Latest Headlines

updated 11:01pm EST

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 ...

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 ...

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 ...

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 ...

Earlier Headlines

Summaries
Headlines

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 ...

New Gene Editing Strategy Could Lead to Treatments for People Born With Inherited Diseases of the Immune System

Oct. 26, 2022 — A fault in cells that form a key part of the immune system can be repaired with a pioneering gene editing technique, finds new research demonstrated in human cells and ...

Gene Mutations in Tumors Impact Radiation Sensitivity

Oct. 20, 2022 — A new study identifies common and rare gene mutations that impact radiation resistance and sensitivity, an important step toward providing more individualized and effective radiotherapy for patients ...

Researchers Identify a Gene as a Potential Target in Treatment-Resistant Brain Cancer Glioblastoma Multiforme

Oct. 19, 2022 — Research led by doctors and scientists have identified a gene that may provide a therapeutic target for the deadly, treatment-resistant brain cancer glioblastoma multiforme ...

Gene Therapy Rapidly Improves Night Vision in Adults With Congenital Blindness

Oct. 11, 2022 — Adults with a genetic form of childhood-onset blindness experienced striking recoveries of night vision within days of receiving an experimental gene ...

Researchers Improve Vehicle for Delivering Gene Therapies to the Central Nervous System

Oct. 10, 2022 — The blood-brain barrier (BBB) is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain ...

Potential of Precision Genome Editing in Treating Inherited Retinal Diseases

Sep. 23, 2022 — Researchers explain how precision genome editing agents have enabled precise gene correction and disease rescue in inherited retinal diseases ...

New Gene Therapy Shows Promise for Treating Eye Condition Affecting Millions Across the Globe

Aug. 24, 2022 — Researchers have developed a new gene therapy approach that shows promise for treating the dry form of Age Related Macular Degeneration (AMD) -- a progressive eye disease that affects up to 10% of ...

Mouse Study Shows Gene Therapy May Correct Creatine Deficiency Disorder

June 6, 2022 — A new study in mice finds that a gene therapy appears to correct a rare creatine deficiency disorder that commonly results in intellectual disabilities, problems with speech, involuntary movements ...

Gene Therapy Could Treat Pitt-Hopkins Syndrome, Proof-of-Concept Study Suggests

May 10, 2022 — Scientists reported on an experimental gene-therapy-like technique to restore the normal activity of the TCCF4 gene deficient in individuals with Pitt-Hopkins ...

Establishment of a Pancreatic Cancer Animal Model Using the Pancreas-Targeted Hydrodynamic Gene Delivery Method

Apr. 22, 2022 — Pancreatic cancer has a significantly poor prognosis; therefore, the development of effective treatments is an unmet clinical need. The major drawback in this field was the lack of useful model ...

DNA Discovery Reveals a Critical 'Accordion Effect' for Switching Off Genes

Apr. 5, 2022 — Researchers have revealed how an 'accordion effect' is critical to switching off genes, in a study that transforms the fundamentals of what we know about gene silencing. The finding expands ...

Novel Nuclear microRNA Is Being Developed for the Treatment of Cardiovascular Disease

Apr. 1, 2022 — A novel angiogenic microRNA drug can be a new option for the treatment of ischemic cardiovascular disease, according to a new study. In the study, the researchers describe a novel nuclear acting ...

An Approach to Treating a Severe Congenital Myopathy

Mar. 29, 2022 — The diagnosis is rare, but devastating -- children with congenital muscle disorders often never learn to walk. Until now, there was no chance of recovery, but researchers are now presenting a ...

Tuesday, April 4, 2023