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.

CHOP researchers unlock 3D structure of crucial protein for cancer therapy

Chimeric Antigen Receptors (CARs) have opened up an exciting new field of therapeutic advancements for rare and difficult-to-treat cancers, as they have the ability to deliver targeted therapies that can kill tumor cells.

1 Dec 2023

Silencing galectin 1 protein shrinks liver cancer tumors in mice

Researchers at UC Davis Comprehensive Cancer Center have shown that inhibiting a specific protein using gene therapy can shrink hepatocellular carcinoma (HCC) in mice.

30 Nov 2023

Novel method can detect contaminants in T-cell cultures within 24 hours

Researchers from Critical Analytics for Manufacturing Personalized-Medicine (CAMP) Interdisciplinary Research Group (IRG) at Singapore-MIT Alliance for Research and Technology (SMART), MIT's research enterprise in Singapore, in collaboration with Singapore Centre for Environmental Life Sciences Engineering (SCELSE) and Massachusetts Institute of Technology (MIT), have developed a novel method capable of identifying contaminants in T-cell cultures within 24 hours.

27 Nov 2023

Synthetic biologists develop versatile platform for targeted drug delivery

In recent years, cell and gene therapies have shown significant promise for treating cancer, cystic fibrosis, diabetes, heart disease, HIV/AIDS and other difficult-to-treat diseases.

27 Nov 2023

Gene therapy could reverse symptoms of rare, hereditary DOOR syndrome

A lot of research has been done over many decades on diseases that are widespread in large parts of the population, such as cancer and heart disease. As a result, treatment methods have improved enormously thanks to long-term research efforts on diseases that affect many people.

23 Nov 2023

Genetic vulnerability of leukemia cells uncovered

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.

22 Nov 2023

Combined application of therapeutic viruses and nanomaterials to revolutionize cancer therapy

Using oncolytic viruses (OVs) in combination with nanomaterials as a novel approach to cancer treatment.

22 Nov 2023

Long-term monitoring of gene therapy patients crucial for safety, study says

Medical research has shown promising results regarding the potential of gene therapy to cure genetic conditions such as sickle cell disease and the findings of this study, published in Nature Medicine, offer important new insights into processes happening in the body after treatment.

16 Nov 2023

Gene therapy for rare diseases: A decade of progress and remaining challenges

A new review article in the peer-reviewed journal Human Gene Therapy summarizes the significant milestones in the development of gene therapy medicinal products that have facilitated the treatment of a significant number of rare diseases.

14 Nov 2023

"Christchurch mutation" can block detrimental effects of Alzheimer’s risk factor

Researchers at Gladstone Institutes have discovered that a rare genetic variant known as the "Christchurch mutation" can block detrimental effects of apolipoprotein E4, the best-established risk factor for the most common form of Alzheimer's disease.

13 Nov 2023

Unlocking the cure for hepatitis B virus: Challenges and opportunities

Hepatitis B virus (HBV) is a significant public health problem, with over 296 million people chronically infected worldwide.

13 Nov 2023

Gene therapy's double-edged sword: Breakthrough treatments face manufacturing and efficacy

Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.

12 Nov 2023

Data, Design, and Discovery: Andrew Buchanan's Impact on Biologics Innovation

Andrew Buchanan on the Future of Biologics and AI

10 Nov 2023

Temperature plays crucial role in virus DNA release and infection

Researchers at Lund University, together with colleagues at the NIST Synchrotron Facility in the USA, have mapped on an atomic level what happens in a virus particle when the temperature is raised.

8 Nov 2023

New gene therapy strategy shows promise for Parkinson's disease

Researchers from the Shenzhen Institute of Advanced Technology (SIAT) of the Chinese Academy of Sciences (CAS) and their collaborators have developed a gene therapy strategy to selectively manipulate Parkinson's disease-affected circuitry and attenuate the core motor symptoms of Parkinson's disease in rodent and nonhuman primate animals.

2 Nov 2023

Science, Innovation, and Xenotransplantation: A Discussion with Dr. Wenning Qin

In this interview, we delve deep into the pioneering world of xenotransplantation and the evolving landscape of gene editing.

2 Nov 2023

Proof-of-concept study holds promise for safer gene therapy techniques for treating immunodeficiencies

Researchers described an approach to edit the recombination-activating gene 2 (RAG2) locus for therapeutic applications.

1 Nov 2023

Sickle cell disease patients willing to accept high risks for gene therapy cure

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, according to a study published today in Blood Advances.

31 Oct 2023

Novel replacement strategy holds promise for the treatment of various genetic disorders

Severe Combined Immunodeficiencies (SCIDs) are a group of debilitating primary immunodeficiency disorders, primarily caused by genetic mutations that disrupt T-cell development. SCID can also affect B-cell and natural killer cell function and counts.

27 Oct 2023

Gene therapy breakthrough: Scientists develop method to deliver larger genes to target cells

Gene therapy currently represents the most promising approach for the treatment of hereditary diseases. Yet despite significant breakthroughs in recent years, there are still a number of hurdles that hinder the wider application of gene therapies.

24 Oct 2023