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.

New curative treatment for sickle cell disease shows promising results in international trial

Vanderbilt University Medical Center (VUMC) researchers are touting data from a multicenter, international phase 2 clinical trial showing a new, curative treatment for sickle cell disease (SCD).

25 Jun 2024

The path to perfection: A deep dive into continuous improvement in quality management

This interview examines the principles of continuous improvement and how they can be applied to enhance QMS.

From DSI, a PLG Company 24 Jun 2024

Stilla Technologies expands U.S. presence through strategic distribution relationship with Avantor and introduces advanced content offerings

Stilla, a pioneer in the field of genomics and digital PCR, is excited to announce its expansion in the United States via a strategic distribution relationship with Avantor, a global leader in life sciences and advanced technology solutions.

From Stilla Technologies 20 Jun 2024

New cellular atlas revolutionizes understanding of spinal cord injury

Scientists at EPFL have achieved a significant research milestone in the field of spinal cord injuries - mapping out the cellular and molecular dynamics of paralysis in unprecedented detail with their open-source project 'Tabulae Paralytica'.

19 Jun 2024

Researchers develop cutting-edge smart bandages for improved wound healing

Chronic wounds, which include diabetic ulcers, surgical wounds, pressure injuries, and other problems, are deadlier than many people realize.

17 Jun 2024

Gyros Protein Technologies introduces Gyrolab Generic Anti-AAV Kit to support gene therapy development

Ready-to-use kit enables detection of binding antibodies against AAV vectors without the need for serotype-specific assays.

From Gyros Protein Technologies AB 17 Jun 2024

Efficient whole-gene insertions can be achieved with improved gene-editing technology

Scientists at the Broad Institute of MIT and Harvard have improved a gene-editing technology that is now capable of inserting or substituting entire genes in the genome in human cells efficiently enough to be potentially useful for therapeutic applications.

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?

In 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

Gene Therapy News and Research

Further Reading

New curative treatment for sickle cell disease shows promising results in international trial

The path to perfection: A deep dive into continuous improvement in quality management

Stilla Technologies expands U.S. presence through strategic distribution relationship with Avantor and introduces advanced content offerings

New cellular atlas revolutionizes understanding of spinal cord injury

Researchers develop cutting-edge smart bandages for improved wound healing

Gyros Protein Technologies introduces Gyrolab Generic Anti-AAV Kit to support gene therapy development

Efficient whole-gene insertions can be achieved with improved gene-editing technology

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

Ultimate guide to the essential tools for the characterization of proteins, polymers and nanoparticles eBook

Evaluating new approaches for rare disease genomics

Expanding access to NMR with the Fourier 80 Duo

Advancing Intracellular Delivery with LumiPore®

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