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.

CHARMs: A new hope for prion disease therapy

Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades.

27 Jun 2024

Targeted gene therapy may reduce AML burden and protect the heart from cardiotoxicity

Researchers from the University of Missouri School of Medicine found that a targeted gene therapy may make acute myeloid leukemia (AML) more sensitive to chemotherapy, while also protecting the heart against toxicity often caused by cancer treatments.

27 Jun 2024

Researchers identify key players in abnormal bone differentiation

A study conducted by researchers at Baylor College of Medicine and collaborating institutions reveals the molecular events leading to osteogenesis imperfecta type V, a form of brittle bone disease caused by a mutation in the gene IFITM5.

27 Jun 2024

Comparative study on preventative SMA therapies shows mixed outcomes

In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or in combination with risdiplam (oral medication) or nusinersen (intrathecal injection) administered before apparent signs of disease emerged.

25 Jun 2024

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

Gene Therapy News and Research

Further Reading

CHARMs: A new hope for prion disease therapy

Targeted gene therapy may reduce AML burden and protect the heart from cardiotoxicity

Researchers identify key players in abnormal bone differentiation

Comparative study on preventative SMA therapies shows mixed outcomes

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

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

The Critical Role of Sample Storage in Drug Discovery and Development

Advancing Neuroscience Research Through Collaboration and Patient-Centered Innovation

The impact of vibration on microscopy

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