Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

CRISPR tool unveils genetic drivers of blood cell maturation

Collaborative research led by investigators at Dana-Farber/Boston Children's Cancer and Blood Disorders Center defines a novel approach to understanding how certain proteins called transcription factors determine which genetic programs will drive cell growth and maturation.

4 Apr 2025

Reprogramming the colon could be a therapeutic strategy for short bowel syndrome

Knocking out a single gene reprograms part of the large intestine to function like the nutrient-absorbing small intestine.

3 Apr 2025

4basebio receives MHRA GMP licence for manufacture and supply of synthetic DNA

4basebio PLC (“4basebio”), a company which develops and commercializes the large-scale manufacture of synthetic DNA as well as nanoparticle delivery solutions, today announced that it has received Good Manufacturing Practice (GMP) certification from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).

From 4basebio PLC 3 Apr 2025

Breakthrough research reveals gut-eye connection in vision loss

Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor cells, ultimately leading to vision loss and blindness.

2 Apr 2025

New drug restores vision by regenerating retinal nerves

Vision is one of the most crucial human senses, yet over 300 million people worldwide are at risk of vision loss due to various retinal diseases.

1 Apr 2025

Scientists make breakthrough in understanding the causes of spina bifida

Scientists at Rady Children's Institute for Genomic Medicine, and the Department of Neurosciences and Pediatrics at the University of California, San Diego, have made a significant breakthrough in understanding the causes of spina bifida, a serious birth defect affecting thousands of newborns each year.

1 Apr 2025

Comprehensive AAV delivery map aids in advancing gene therapy

Gene therapy, a technique that is revolutionizing the treatment of multiple genetic conditions, including eye and muscle diseases and blood disorders, requires efficient and specific delivery of the genetic material to the tissue and cell type of interest.

1 Apr 2025

Transforming genetic deafness treatment with base editing

Congenital hearing loss refers to impaired auditory function that occurs due to genetic causes. GJB2 is the gene responsible for approximately half of all cases of hereditary hearing loss.

27 Mar 2025

Yale study unlocks potential of lupus-related antibody to target cold tumors

Yale scientists have discovered a promising way to trigger immune responses against certain tumors, using a lupus-related antibody that can slip, undetected, into "cold" tumors and flip on an immune response that has been turned off by cancer.

25 Mar 2025

Breakthrough DNA delivery system could revolutionize chronic disease treatment

A breakthrough in safely delivering therapeutic DNA to cells could transform treatment for millions suffering from common chronic diseases like heart disease, diabetes, and cancer.

25 Mar 2025

Eye drops show potential to slow progression of human degenerative retinal diseases

Treatment shows potential to slow the progression of human degenerative eye diseases, including retinitis pigmentosa.

21 Mar 2025

In the Thymus We Trust: ProTcell Therapy & the Future of Allogeneic Immunotherapy

In this interview, News Medical speaks with Olivier Negre, Chief Scientific Officer at Smart Immune, about how immunotherapy is being revolutionized.

From Hanson Wade Group - 7th Allogeneic Cell Therapies Summit 18 Mar 2025

New insights into ADAR1 mechanisms could revolutionize autoimmune and cancer therapies

A research team led by Rice University's Yang Gao has uncovered new insights into the molecular mechanisms of ADAR1, a protein that regulates ribonucleic acid (RNA) induced immune responses.

17 Mar 2025

Clinical trial tests herpes virus-based therapy for neurogenic bladder in people with spinal cord injury

A modified herpes virus that targets spinal cord nerve cells to treat neurogenic bladder in people with spinal cord injury is underway in a first-in-human clinical trial by UTHealth Houston at TIRR Memorial Hermann.

12 Mar 2025

Study reveals key insights for preventing cytomegalovirus spread to fetuses

A new Weill Cornell Medicine and Oregon Health & Science University co-authored study provides critical insight for the development of a vaccine that can more effectively block the spread of cytomegalovirus, or CMV, across the placenta to babies before they are born.

12 Mar 2025

Many individuals with Niemann-Pick disease lack neutralizing antibodies against AAV2 or AAV9

A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies against either adeno-associated virus (AAV) 2 or AAV9.

10 Mar 2025

Pioneering the world’s first CRISPR medicine for sickle cell disease

When Vijay Sankaran was an MD-PhD student at Harvard Medical School in the mid-2000s, one of his first clinical encounters was with a 24-year-old patient whose sickle cell disease left them with almost weekly pain episodes.

3 Mar 2025

Study demonstrates successful gene therapy for maple syrup urine disease

A study led by UMass Chan researchers demonstrated that a gene therapy to correct a mutation that causes maple syrup urine disease (MSUD) prevented newborn death, normalized growth, restored coordinated expression of the affected genes and stabilized biomarkers in a calf as well as in mice.

28 Feb 2025

New deep-learning framework improves spatial transcriptomics analysis

Biological tissues are made up of different cell types arranged in specific patterns, which are essential to their proper functioning.

27 Feb 2025

New gene therapy strategy could cure COPA Syndrome

Scientists compared five families and multiple generations to find a gene variant that prevents COPA Syndrome and opens the door to a new gene therapy for the condition.

27 Feb 2025