Gene Therapy News and Research

RSS

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.

New US headquarters opened by mass photometry pioneers Refeyn

Refeyn, the company behind pioneering mass photometry technologies, has officially opened its new 10,000 sq. ft. US Headquarters and Customer Interaction Center in Waltham’s Biotech Hub in Greater Boston, Massachusetts.

From Refeyn 10 Jun 2024

Novel therapeutic approach shows promise for frontotemporal dementia in pre-clinical trials

Pre-clinical trials: A new therapeutic approach for the severe condition uses modified viruses to replace a missing protein in the brain.

7 Jun 2024

Gene therapy trial: restored hearing in children with hereditary deafness

The safety and effectiveness of binaural adeno-associated virus 1 (AAV1)-human otoferlin (hOTOF) therapy in five children with autosomal recessive deafness 9 (DFNB9).

7 Jun 2024

InDevR and Sino Biological team up to deliver multiplexed analytical solutions for mRNA vaccine and cell & gene therapy

InDevR, Inc., a leader in analytical solutions for vaccine in-process and quality control testing, and Sino Biological, Inc., a biotechnology company listed on the Shenzhen stock exchange subsidiary ChiNext specializing in biological research reagents and related technical contract research services, today announced a new partnership to speed development of multiplexed assays for a broad range of vaccine, mRNA therapies and cell and gene therapy applications.

From Sino Biological Inc. 7 Jun 2024

Novel technique maps enhancer-gene interactions in single cells

The human genome contains about 23,000 genes, but only a fraction of those genes are turned on inside a cell at any given time.

5 Jun 2024

New bilateral gene therapy shows promising results in treating genetic hearing loss

A novel gene therapy designed to target a form of inherited deafness restored hearing function in five children who were treated in both ears.

5 Jun 2024

Foam technology revolutionizes gene therapy, boosting efficiency and safety

Liquid foam gene delivery significantly enhances transfection efficiency and safety, offering a cost-effective alternative to conventional methods.

3 Jun 2024

Miniaturized CRISPR protein opens door for gene therapy with adeno-associated virus

Researchers have developed a novel version of a key CRISPR gene-editing protein that shows efficient editing activity and is small enough to be packaged within a non-pathogenic virus that can deliver it to target cells.

30 May 2024

Foam-based delivery system could revolutionize gene therapies

Foam mixed with medications is already used to treat conditions such as varicose veins, hemorrhoids, wounds on the skin and even hair loss.

29 May 2024

Researchers decode spatial structure of prime editor

Joint research led by Yutaro Shuto, Ryoya Nakagawa, and Osamu Nureki of the University of Tokyo determined the spatial structure of various processes of a novel gene-editing tool called "prime editor."

29 May 2024

Cellular Origins and Cell and Gene Therapy Catapult collaborate to demonstrate universal automation of CGT manufacturing

Cellular Origins, a TTP Company focused on enabling scalable, cost-effective, and efficient manufacture of cell and gene therapies (CGTs), and the Cell and Gene Therapy Catapult (CGT Catapult), an independent innovation and technology organisation specializing in the advancement of the cell and gene therapy industry, today announced a collaboration to demonstrate universal automation of CGT manufacturing.

29 May 2024

Why use light scattering to analyze proteins and viral vectors?

n this interview, Dr. Michelle Chen, Senior Director of Analytical Sciences at Wyatt Technology, talks to NewsMedical about how to use light scattering techniques to analyze proteins for their multi-attribute quantification (MAQ).

From Waters | Wyatt Technology 29 May 2024

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

Gene Therapy News and Research

Further Reading

New US headquarters opened by mass photometry pioneers Refeyn

Novel therapeutic approach shows promise for frontotemporal dementia in pre-clinical trials

Gene therapy trial: restored hearing in children with hereditary deafness

InDevR and Sino Biological team up to deliver multiplexed analytical solutions for mRNA vaccine and cell & gene therapy

Novel technique maps enhancer-gene interactions in single cells

New bilateral gene therapy shows promising results in treating genetic hearing loss

Foam technology revolutionizes gene therapy, boosting efficiency and safety

Miniaturized CRISPR protein opens door for gene therapy with adeno-associated virus

Foam-based delivery system could revolutionize gene therapies

Researchers decode spatial structure of prime editor

Cellular Origins and Cell and Gene Therapy Catapult collaborate to demonstrate universal automation of CGT manufacturing

Why use light scattering to analyze proteins and viral vectors?

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

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

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

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

Novel gene-delivery vehicle shows promise for treating brain disorders

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

Experimental gene therapy shows promise for treating herpes infections

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

Discover how SciY empowers scientists

Why use light scattering to analyze proteins and viral vectors?

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

Latest News