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.

Novel drug holds promise for treating Duchenne muscular dystrophy

A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration.

16 Dec 2024

Targeted gene therapy shuts down cancer cells' energy centers

Researchers are shining a light on cancer cells' energy centers – literally – to damage these power sources and trigger widespread cancer cell death.

14 Dec 2024

New protein platform enhances cancer immunotherapy

Researchers from Children's Hospital of Philadelphia (CHOP) and Stanford University have revealed the molecular structure of TRACeR-I, a protein platform for reprogramming immune responses. A better understanding of its structure may help optimize designs for the platform, which can be used to develop cancer treatments by either directly modifying immune cells or by creating proteins that help immune cells locate cancer cells. The findings were published today by the journal Nature Biotechnology.

13 Dec 2024

Gene therapy to prevent stillbirth and premature delivery developed

Researchers investigate the effectiveness of placental nano-based insulin-like 1 growth factor gene therapy to correct fetal growth restriction.

12 Dec 2024

Scientists identify potential therapeutic targets for diabetic kidney disease

New potential therapeutic targets have been identified for diabetic kidney disease (DKD) - the leading cause of kidney failure in the world - that could see patients treated with new gene and drug therapies preventing the disease's progression into end stage kidney failure.

10 Dec 2024

Promising gene therapy could transform heart failure treatment

A new gene therapy can reverse the effects of heart failure and restore heart function in a large animal model.

10 Dec 2024

Polypurine hairpins found to effectively halt SARS-CoV-2 replication

A study led by the University of Barcelona and the Spanish National Research Council's Institute for Advanced Chemistry of Catalonia (IQAC - CSIC) presents a new therapeutic tool capable of inhibiting the proliferation of the SARS-CoV-2 virus that causes COVID-19.

3 Dec 2024

First U.S. trial uses non-viral CRISPR to correct sickle cell mutation

UCSF Benioff Children's Hospital Oakland is enrolling patients in an innovative clinical trial that seeks to cure sickle cell disease.

26 Nov 2024

ESGCT Conference Takeaways: A specialty laboratory perspective

Discover the latest innovations in cell and gene therapy via Cerba Research's highlights of ESGCT.

From Cerba Research 22 Nov 2024

From crude lysate to purity with high-precision AAV capsid measurement with Bio-Rad's new ddPCR Kit

Accurately quantify AAV capsids directly from crude lysates with Bio-Rad's new ddPCR kit—offering reliable, efficient, and precise measurement to streamline your research workflows.

From Bio-Rad Laboratories 19 Nov 2024

Stilla Technologies with Source BioScience brings advanced genomics solutions to the UK market

Stilla Technologies, a global leader in digital PCR (dPCR) solutions, has announced a strategic partnership with Source BioScience, a leading UK-based genomics service provider.

From Stilla Technologies 19 Nov 2024

StitchR technology delivers large genes for muscular dystrophy treatment

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

14 Nov 2024

UAB researchers reverse liver disease in mice with gene therapy

UAB researchers have reversed metabolic dysfunction-associated steatohepatitis (MASH) in mouse models. MASH is a severe liver disease associated with obesity and type 2 diabetes that affects more than 40 million people.

13 Nov 2024

MaxCyte celebrates 25 years of innovation driving cell engineering-based therapeutics

MaxCyte, Inc., a leading, cell-engineering focused company providing enabling platform technologies to advance the discovery, development and commercialization of next-generation cell therapeutics, marks the 25th anniversary of its founding this year.

From MaxCyte, Inc. 13 Nov 2024

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Professor Nancy Ip discusses her groundbreaking neuroscience research, focusing on neurotrophic factors and innovative Alzheimer's disease treatment approaches.

13 Nov 2024

Utilizing process Raman in optimizing cultivated meat production

Learn about the usage of process raman spectroscopy in the optimization of bioreactor monitoring and then improvement of cultivated meat production.

From Thermo Fisher Scientific – Pharmaceutical and Biopharmaceutical Solutions 13 Nov 2024

New approach reinvigorates exhausted T cells to improve tumor control

As cancer cells grow, they pump out metabolic byproducts such as lactic acid into the tumor microenvironment. Exhausted T cells - which have lost their cancer-fighting oomph - consume this lactic acid, which further saps their energy, according to new research from the University of Pittsburgh and UPMC Hillman Cancer Center.

8 Nov 2024