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 mechanisms to enable regeneration of injured nerve fibers

Injuries to nerve fibers in the brain, spinal cord, and optic nerves usually result in functional losses as the nerve fibers are unable to regenerate.

23 Aug 2019

Research examines role of Nicotinamide in preventing retinal cell damage caused by glaucoma

Fight for Sight is funding researchers at Cardiff University to examine the extent to which dietary supplementation of Nicotinamide – a form of vitamin B3 – can be used to reduce, and even prevent, retinal damage caused by glaucoma.

21 Aug 2019

California clamps down on amateur use of gene-editing technologies

California state officials have passed the first law to prohibit genetic biohacking in an effort to regulate at-home gene editing.

20 Aug 2019

Discover the latest advances in neuromuscular therapeutics at NMD 2019

The 2nd Neuromuscular Drug Development Summit, or NMD, will be held in Boston, MA, from October 23-25,2019.

From Hanson Wade 19 Aug 2019

Tooth repair using stem cells may soon be a reality

A team of researchers including those from Biotechnology Center of the TU Dresden (BIOTEC), have found that stem cells could be used for several forms of tissue engineering including tooth repair.

13 Aug 2019

Antibody may help the immune system fight sepsis-related fungal infections

An antibody that blocks the "programmed cell death" pathway may help the immune system fight off sepsis-related fungal infections, according to animal studies reported in SHOCK: Injury, Inflammation, and Sepsis: Laboratory and Clinical Approaches, Official Journal of the Shock Society.

12 Aug 2019

ISCT expresses concern about companies offering unproven T-cell preservation services

ISCT, the International Society for Cell and Gene Therapy, the global professional society of clinicians, researchers, regulatory specialists, technologists and industry partners in the cell and gene therapy sector, today announces its objection to linking the benefits of cellular immunotherapy, in particular Chimeric Antigen Receptor (CAR) T-cell therapy, with recent market offerings for third party cryopreserving and banking of T-cells for future therapeutic use.

8 Aug 2019

Genome editing strategy opens new perspectives in the treatment of cystic fibrosis

The approach adopted by the team of the University of Trento, led by Anna Cereseto, opens new perspectives in the treatment of cystic fibrosis, a genetic disease for which no cure is currently available.

7 Aug 2019

Statement related to data inaccuracies with newly approved gene therapy

As a public health agency, we believe that it is critical to facilitate the development of innovative safe and effective medical products, like the cellular and gene therapy products that have shown enormous potential to treat previously untreatable diseases.

7 Aug 2019

New CRISPR tool enhances RNA editing

CRISPR-based tools helped revolutionize the ability to target genetic mutations linked to disease development. Such tools focus on using the enzymes Cas9 and Cas12 to target DNA, and Cas13 to target RNA. Now, scientists used a new CRISPR platform that expands the capabilities of RNA editing.

29 Jul 2019

New biomimetic culture system enables more extensive testing of drugs and gene therapies

Researchers at the University of Louisville have developed an easily reproducible system that enables them to keep slices of human hearts alive for a longer period of time, allowing more extensive testing of new drugs and gene therapies.

26 Jul 2019

New vaccine shows promise against toughest HIV strains

A new study reports successful immunization against a so-far invincible “death star” strain of an HIV-related laboratory virus (SIVmac239) and another tough strain. The new approach also achieves long-term protection with a single dose, showing that gene therapy could allow broad and durable protection, based on a modified form of the CD4 protein that allows HIV entry into human cells.

24 Jul 2019

Measuring pupil dilation and heart rate using AI could enable earlier diagnosis of autism

Autism and other neurodevelopmental disorders often aren't diagnosed until a child is a few years of age, when behavioral interventions and speech/occupational therapy become less effective.

24 Jul 2019

Researchers explore stem cells for answers to how fragile X syndrome develops

The lab of Yongchao Ma, PhD, from Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago, discovered how the genetic defect in fragile X syndrome – a type of autism – delays production of neurons (nerve cells) at a critical time in the embryo's brain development.

24 Jul 2019

BIA Separations selected to support AveXis’ gene therapy pipeline

BIA Separations, a leading biochromatography development and manufacturing company, today announced it is collaborating with AveXis, a Novartis company, to enhance the commercial purification process for its gene therapy pipeline, beginning with Zolgensma (onasemnogene abeparvovec-xioi), the first U.S. Food and Drug Administration-approved gene therapy indicated for the treatment of pediatric patients with spinal muscular atrophy (SMA) who are less than two years of age.

22 Jul 2019

Novel insight into microRNA function can be crucial for the development of gene therapy

Scientists at the University of Eastern Finland and the University of Oxford have shown that small RNA molecules occurring naturally in cells, i.e. microRNAs, are also abundant in cell nuclei.

18 Jul 2019

Research opens possibility of developing single-dose gene therapy for inherited arrhythmias

Researchers at Boston Children's Hospital report creating the first human tissue model of an inherited heart arrhythmia, replicating two patients' abnormal heart rhythms in a dish, and then suppressing the arrhythmia with gene therapy in a mouse model.

17 Jul 2019

Don't Miss the Blood-Brain Barrier Drug Delivery (B3DD) Summit this August

The B3DD Summit will be held in Boston from August 13-15, 2019. This news story provides an overview of what you can expect at this year's conference.

From Blood-Brain Barrier Delivery Summit (B3DD) 17 Jul 2019

Gene modulation goes wireless hacking the "boss gene"

Researchers from the University at Buffalo have successfully used technology to wirelessly tweak the functioning of a gene FGFR1. This gene is vital in the growth of embryos into adults. Their experiments were successful in the lab grown brain tissues and shows potential in therapeutics.

16 Jul 2019

Nanoparticles used to deliver CRISPR gene editing tools into the cell

Researchers from Tufts University and the Chinese Academy of Sciences have developed a novel method to effectively deliver the CRISPR/Cas9 (clustered regularly interspaced short palindromic repeat (CRISPR) associated protein 9) gene editing tools into the liver for genetic studies.

15 Jul 2019