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.

Changing the landscape of R&D to build clinical success from the ground up

In this interview, NewsMedical talks to Amanda Jones at Revvity about the strategies and solutions available to revolutionize the landscape of R&D in clinical research.

From Revvity 28 May 2024

Two new studies offer insights into the molecular biology of neuropsychiatric disease

Working under the umbrella of the PsychENCODE Consortium, the mental health research project established in 2015 by the National Institutes of Health, a team of Mount Sinai scientists has uncovered important new insights into the molecular biology of neuropsychiatric disease through two new studies published in a special issue of Science on Friday, May 24.

24 May 2024

Novel catalog of gene-isoform variation unveiled in the developing human brain

A team led by researchers at UCLA and the University of Pennsylvania has produced a first-of-its kind catalog of gene-isoform variation in the developing human brain.

23 May 2024

Non-viral gene therapy offers hope for chronic low back pain sufferers

A novel non-viral gene therapy for discogenic back pain (DBP).

20 May 2024

Novel gene-delivery vehicle shows promise for treating brain disorders

In an important step toward more effective gene therapies for brain diseases, researchers from the Broad Institute of MIT and Harvard have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

16 May 2024

Investigational drug shows promise in increasing oxygen uptake for patients with heart condition

People with a common heart condition were able to use significantly more oxygen while exercising after taking an investigational drug in an international clinical trial, according to a study published today in the New England Journal of Medicine.

14 May 2024

Experimental gene therapy shows promise for treating herpes infections

Researchers at Fred Hutch Cancer Center have found in pre-clinical studies that an experimental gene therapy for genital and oral herpes removed 90% or more of the infection and suppressed how much virus can be released from an infected individual, which suggests that the therapy would also reduce the spread of the virus.

13 May 2024

Genetic variant increases heart failure risk and decreases longevity in Black Americans

Researchers at Brigham and Women’s Hospital and Duke University showed that a genetic variant, present in 3-4% of self-identified Black individuals in the U.S., increases the risk for both heart failure and death and contributes to significant decreases in longevity at the population level.

11 May 2024

CRISPR-based gene editing shows promise in treating rare form of blindness

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published in the New England Journal of Medicine.

6 May 2024

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

Results from a groundbreaking clinical trial of CRISPR gene editing in 14 individuals with a form of inherited blindness show that the treatment is safe and led to measurable improvements in 11 of the participants treated.

6 May 2024

Lab to plant: Scaling up API processes with Dr. James Mencel's guidance

In this interview, NewsMed talks to Dr. James Mencel's Guidance about Scaling Up API Processes.

From DSI, a PLG Company 6 May 2024

GenE-HumDi's strategy for involving young scientists in cutting-edge research

COST Actions are meant to be inclusive which means ensuring a geographic spread of participants, encouraging gender balance, and involving young researchers.

3 May 2024

ELRIG UK announces Profs Ijeoma Uchegbu and Marcus Schindler as keynote speakers at Drug Discovery 2024

Drug Discovery 2024: Connecting Minds, Transforming Science, Empowering Community will take place at ExCeL London from 2–3 October.

From ELRIG (UK) Ltd. 2 May 2024

Electric pulses boost gene therapy uptake in liver cells

In an effort to improve delivery of costly medical treatments, a team of researchers in electrical engineering at the University of Wisconsin–Madison has developed a stimulating method that could make the human body more receptive to certain gene therapies.

30 Apr 2024

ZIP7: A novel target for treating degenerative diseases associated with misfolded proteins

Proteins are the workhorses of life. Organisms use them as building blocks, receptors, processors, couriers and catalysts.

30 Apr 2024

CASGEVY gene therapy eliminates vaso-occlusive crises in sickle cell patients

In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

24 Apr 2024

Novel immunotherapy shows promise for high-risk sarcomas: HEROS 2.0 trial results published

Researchers at Texas Children’s Cancer Center and the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children’s Hospital and Houston Methodist published results of a phase I clinical trial of a novel immunotherapy for high-risk sarcomas in the journal Nature Cancer.

24 Apr 2024

Top pharmaceutical company evaluates Optimer for precision liver medicine

Optimer delivery vehicle has been shipped to a pharma partner for external evaluation.

From Aptamer Group 22 Apr 2024

Researchers receive NIH grant to help develop gene therapy for HIV

Researchers at Washington University School of Medicine in St. Louis have received a $6.2 million grant from the National Institutes of Health (NIH) to develop a gene therapy that would modify the immune system's B cells to spur them to produce broadly neutralizing antibodies against HIV.

18 Apr 2024

New platform can deliver gene therapy precisely to specific areas of the brain

A novel approach to gene therapy is improving lives in ways once thought impossible. Researchers at The Ohio State University Wexner Medical Center and College of Medicine have developed a new platform to deliver the gene therapy precisely to specific areas of the brain.

18 Apr 2024