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.

Northwestern scientists receive awards totaling nearly $18 million for Parkinson's disease research

Northwestern University scientists have received two awards totaling nearly $18 million for Parkinson's disease research from The ASAP Collaborative Research Network, a program of the Aligning Science Across Parkinson's (ASAP) initiative being implemented through The Michael J. Fox Foundation.

27 Oct 2021

Researchers receive $8.9 million grant to study the gut-brain connection in Parkinson’s disease

Dr. Michael Kaplitt, a professor of neurological surgery at Weill Cornell Medicine and a longtime leader in developing cutting-edge surgical therapies for movement disorders, leads a team that has been awarded a three-year, $8.9 million grant from the Aligning Science Across Parkinson's (ASAP) initiative.

26 Oct 2021

Safer, more effective therapies could help fight the obesity epidemic

Novel obesity treatments such as modulation of the gut microbiome and gene therapy are underutilized and could help fight the obesity epidemic, according to a new manuscript published in the Endocrine Society's journal, Endocrine Reviews.

26 Oct 2021

MIP Diagnostics to support the development of challenging affinity reagents with new Molecular Imprinting Epitope Discovery

MIP Diagnostics has today announced the launch of its novel Molecular Imprinting Epitope Discovery Service.

26 Oct 2021

Gene therapy strategy against Angelman syndrome shows early promise

Scientists at the UNC School of Medicine have reported in the journal JCI Insight encouraging early tests of a gene therapy strategy against Angelman syndrome, a neurodevelopmental disorder that features poor muscle control and balance, hard-to-treat epilepsy, and intellectual disabilities.

22 Oct 2021

Researchers identify new gene variants that may cause inherited retinal dystrophies

An international team of researchers, led by scientists at University of California San Diego and Shiley Eye Institute at UC San Diego Health, has broadened and deepened understanding of how inherited retinal dystrophies (IRDs) affect different populations of people and, in the process, have identified new gene variants that may cause the diseases.

20 Oct 2021

New technology streamlines the development of gene therapy for genetic blinding disorders

A novel computational platform developed by researchers from the University of Pittsburgh School of Medicine identifies top-performing viral vectors that could deliver gene therapies to the retina with maximum efficiency and precision.

19 Oct 2021

Researchers reveal the mechanism of artificial chromosome formation in C. elegans' embryos

A research team led by Dr Karen Wing Yee YUEN, Associate Professor from the School of Biological Sciences at the University of Hong Kong (HKU), revealed the mechanism of artificial chromosome (AC) formation in the embryos of the model organism Caenorhabditis elegans, a 1-mm long, transparent nematode.

18 Oct 2021

Researchers report long-lasting effects of gene therapy in children with deadly immune disorder

Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder.

18 Oct 2021

Devastating genetic disease in children may be treatable in adulthood

A devastating genetic disease called CDKL5 deficiency disorder (CDD), which strikes in early childhood, may be significantly treatable even in adulthood, a new study from the Perelman School of Medicine at the University of Pennsylvania suggests.

15 Oct 2021

Researchers identify neurons and signaling pathways that make sexually receptive females more active

Scientists have known for a century that female animals become more active just as they are about to ovulate, a behavior that evolved to enhance their chances of mating when they are fertile.

15 Oct 2021

Developing New Technology for the Encapsulation of mRNA-Based Vaccines in Lipid Nanoparticles (LNPs)

NewsMed speaks to KNAUER about the company’s background, its range of specialisms and its pioneering work to facilitate the largescale production of LNPs while meeting the stringent regulations and exacting demands of the biopharmaceutical industry.

From KNAUER Wissenschaftliche Geräte GmbH 14 Oct 2021

Drug developed at Duke is approved by FDA as only therapy for congenital athymia

The phone rang into the pediatric unit that Louise Markert, M.D., Ph.D., was staffing as a junior faculty member in 1991. Had she not answered, there would have been little hope for the more than one hundred patients who were subsequently treated.

11 Oct 2021

Study supports efficacy and safety of lung-selective nanotherapy against SARS-CoV-2 infection

A new preprint shows the potential role of a host protease cathepsin L inhibitor, which mediates viral entry into the host cell by its effect on the viral spike protein.

8 Oct 2021

Australian researchers hope to switch on sight with new gene therapy

Australian researchers are developing a new gene therapy which they hope will ‘switch on sight’ by regenerating light-sensing photoreceptor cells in the retina at the back of the eye.

7 Oct 2021

New understanding of cellular defects related to Cystic Fibrosis may pave the way for treatments

A team in the College of Medicine led by Drs. Juan Ianowski (PhD) and Julian Tam (MD) found that sodium transport is abnormal in lungs with CF.

6 Oct 2021

Researchers identify mechanism that can lead to deafness in rare Norrie disease

A team of Mass Eye and Ear Scientists led by Albert Edge, PhD, working with research fellow Yushi Hayashi, MD, PhD, has identified the mechanism that can lead to deafness in the rare syndrome, Norrie disease.

5 Oct 2021

Restoring visual function after stroke with gene therapy

Most strokes happen when an artery in the brain becomes blocked. Blood flow to the neural tissue stops, and those tissues typically die. Because of the locations of the major arteries in the brain, many strokes affect motor function.

3 Oct 2021

Gyros Protein Technologies introduces Gyrolab AAV9 Titer Kit to support AAV-based cell and gene therapy production

Gyros Protein Technologies AB today announced the introduction of Gyrolab® AAV9 Titer Kit for rapid determination of titer in AAV9 vector-based cell and gene therapy manufacturing.

From Gyros Protein Technologies AB 30 Sep 2021

Supercoiling and looping can transmit mechanical stress along the DNA backbone, research shows

When people think of DNA, they visualize a string-like double helix structure. In reality, the DNA double helix in cells is supercoiled and constrained into loops. This supercoiling and looping are known to influence every aspect of DNA activity, but how this happens has not been clear.

28 Sep 2021

Gene Therapy News and Research

Further Reading

Northwestern scientists receive awards totaling nearly $18 million for Parkinson's disease research

Researchers receive $8.9 million grant to study the gut-brain connection in Parkinson’s disease

Safer, more effective therapies could help fight the obesity epidemic

MIP Diagnostics to support the development of challenging affinity reagents with new Molecular Imprinting Epitope Discovery

Gene therapy strategy against Angelman syndrome shows early promise

Researchers identify new gene variants that may cause inherited retinal dystrophies

New technology streamlines the development of gene therapy for genetic blinding disorders

Researchers reveal the mechanism of artificial chromosome formation in C. elegans' embryos

Researchers report long-lasting effects of gene therapy in children with deadly immune disorder

Devastating genetic disease in children may be treatable in adulthood

Researchers identify neurons and signaling pathways that make sexually receptive females more active

Developing New Technology for the Encapsulation of mRNA-Based Vaccines in Lipid Nanoparticles (LNPs)

Drug developed at Duke is approved by FDA as only therapy for congenital athymia

Study supports efficacy and safety of lung-selective nanotherapy against SARS-CoV-2 infection

Australian researchers hope to switch on sight with new gene therapy

New understanding of cellular defects related to Cystic Fibrosis may pave the way for treatments

Researchers identify mechanism that can lead to deafness in rare Norrie disease

Restoring visual function after stroke with gene therapy

Gyros Protein Technologies introduces Gyrolab AAV9 Titer Kit to support AAV-based cell and gene therapy production

Supercoiling and looping can transmit mechanical stress along the DNA backbone, research shows

Revolutionizing Life Science: An Interview with SCIEX on ASMS, the SCIEX 7500+ System, and AI-Driven Quantitation

From Discovery Biology to ELRIG Chair

Breathing New Life into Diagnostics: Plasmion's SICRIT Technology

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