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.

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

Scientists develop gene therapy approach for rare paralyzing disease

The 50 families stretch from the Netherlands and the United Kingdom to the United States and China. Each family has a child who is paralyzed from a mutation in a single gene named Contactin-Associated Protein 1 (Cntnap1).

20 Oct 2023

Research reveals new way viruses suppress the CRISPR-Cas immune systems of bacteria

A microscopic discovery will not only enable scientists to understand the microbial world around us but could also provide a new way to control CRISPR-Cas biotechnologies.

18 Oct 2023

FAST joins with University of Pennsylvania to develop investigational AAV gene therapy for Angelman syndrome

The Foundation for Angelman Syndrome Therapeutics (FAST) announced today that the non-profit organization has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome (AS).

17 Oct 2023

OrganaBio launches new division to meet growing demand for decentralized clinical trial support

OrganaBio, the Contract Technology Development and Manufacturing Organization (CTDMO) specializing in solutions for cell and immunotherapy development, today announces the official launch of Cell Processing and Cryopreservation (CPC) Services, a new division of OrganaBio.

12 Oct 2023