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.

A powerful tool for separation and sizing of virus mixtures

Postnova Analytics has published an applications report that presents data from a collaboration with the Analytical Development group at Biogen Inc. on separation of a virus mixture using Asymmetrical Flow Field-Flow Fractionation and measurement of their radius of gyration.

From Postnova Analytics 27 Aug 2020

Study offers hope for treatment of Krabbe disease

In one out of 100,000 infants, a mutation in the GALC gene causes an incurable, always fatal disorder known as infantile Krabbe disease, or globoid cell leukodystrophy. Most children with the condition die before they turn 2.

26 Aug 2020

Study examines safety and early outcomes of gene therapy for spinal muscular atrophy

In May 2019, the U.S. Food and Drug Administration approved a gene replacement therapy for the inherited, progressive neuromuscular disease 5q-linked spinal muscular atrophy.

25 Aug 2020

Cobra Biologics successfully produced master cell banks for CombiGene’s gene therapy

Cobra Biologics, part of the Cognate BioServices family, an international CDMO manufacturer of biological materials and pharmaceuticals, and CombiGene AB, the leading gene therapy company in the Nordic region, today announced that Cobra has successfully produced master cell banks for the three plasmids used as starting material for CombiGene’s gene therapy CG01.

21 Aug 2020

Using engineered off-the-shelf therapeutic T cells to fight cancer

Personalized cancer treatments are no longer just options of the future. In the past few years, researchers have made significant progress in 'teaching' the body's immune T cells to recognize and kill specific cancer cells, and human clinical trials have shown that this approach can successfully eliminate tumors.

20 Aug 2020

New treatment approach strengthens motor function and muscle of aged mice

In an aging society, one of the most important and urgent tasks of scientific research is to counteract the decline in motor function and muscle weakness that accompanies the aging process.

17 Aug 2020

Deciphering the largest CRISPR system

Professor Guillermo Montoya spoke to News-Medical on his research that involved visualizing the largest and most complex CRISPR system.

17 Aug 2020

Nanoparticle carriers expand the effectiveness of gene therapies for human eye diseases

NIBIB-funded researchers have created nanoparticles for successful gene therapy of a mouse model of macular degeneration.

15 Aug 2020

Engineered capsids for improved gene delivery to the retina and cornea

A rational design approach created novel variants of adeno-associated viral (AAV) capsids.

13 Aug 2020

Neil Bell appointed as Chief Development Officer of Avacta Life Sciences

Avacta Group plc, the developer of Affimer biotherapeutics and reagents, is pleased to announce the appointment of Neil Bell as Chief Development Officer of Avacta Life Sciences with immediate effect. Neil will be responsible for late stage pre-clinical and early clinical development of Avacta’s pipeline of pre|CISION pro-drugs and Affimer immunotherapies.

From Avacta Life Sciences Ltd. 13 Aug 2020

New techniques help replace malfunctioning microglia associated with neurological disorders

Researchers at Fudan University in Shanghai, China have developed three different techniques that successfully replace almost all malfunctioning microglia - each technique with its own advantage in application

12 Aug 2020

COMBAT study shows how pancreatic cancer patients can benefit from immunotherapy

Working with an international team of researchers, HonorHealth Research Institute and the Translational Genomics Research Institute, an affiliate of City of Hope, were instrumental in one of the first clinical trials showing how pancreatic cancer patients can benefit from immunotherapy, according to a four-year study published in a premier scientific journal, Nature Medicine.

11 Aug 2020

Study may help develop treatment for age-related pulmonary fibrosis

Idiopathic pulmonary fibrosis is a potentially lethal disease for which there is currently no cure and that is associated with certain mutations or advanced age.

11 Aug 2020

Gene therapy to the inner retina prevents blindness in mouse model of CLN3 Batten disease

Gene therapy to the inner retina prevented blindness in a mouse model of the neuro-degenerative disorder CLN3 Batten disease.

10 Aug 2020

New approach improves SCA1 symptoms in animal models

Research has shown that a mutation in the ATAXIN-1 gene leads to accumulation of Ataxin-1 (ATXN1) protein in brain cells and is the root cause of a rare genetic neurodegenerative disease known as spinocerebellar ataxia type 1 (SCA1).

7 Aug 2020

WVU RNI expands pioneering treatment for Alzheimer’s to open the blood-brain barrier

A hospital nurse for 33 years, Nanette Miller would call her husband Frank at the end of each shift to let him know she was coming home. On Dec. 7, 2018, the phone call came with a somber declaration: "I can't do this anymore."

6 Aug 2020

NCI gives 'exceptional' rating to Penn's Abramson Cancer Center

The National Cancer Institute has once again rated the Abramson Cancer Center of the University of Pennsylvania as "exceptional," the highest possible rating for an NCI-designated comprehensive cancer center.

4 Aug 2020

Penn researchers identify genetic driver of Charcot-Marie-Tooth disease

Inherited mutations in a gene that keeps nerve cells intact was shown, for the first time, to be a driver of a neuropathy known as Charcot-Marie-Tooth (CMT) disease.

3 Aug 2020

Thermo Fisher Scientific launches industry-leading, cGMP-compatible CTS Series Laboratory Equipment

Thermo Fisher Scientific announces the launch of an industry-leading product, compliance and services portfolio, compatible with current Good Manufacturing Practice and designed to enable rapid start-up of clinical and commercial cell and gene therapy manufacturing.

From Thermo Fisher Scientific Inc. 30 Jul 2020

Avacta and Daewoong Pharmaceutical expand partnership to include COVID-19 neutralising Affimer therapy

Avacta Group plc, is pleased to announce an expansion of its collaboration and license agreement with Daewoong Pharmaceutical Co.Ltd and AffyXell Therapeutics.

29 Jul 2020