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.

Nanoparticles mimicking flu viruses to deliver large biological drug molecules

Nanoengineers from the University of California San Diego recently created a new and potentially efficient means to deliver messenger RNA (mRNA) into cells.

2 Dec 2021

CRISPR introduces potentially dangerous changes in cells, reveals research

New research revealed that gene editing, particularly gene knockout, with CRISPR-Cas9 can support cells with mutated gene forms associated with cancer.

15 Nov 2021

Researchers employ hepatic organoids for disease modeling

Associate Professor Tamer Önder created and analyzed the hepatic organoid culture system utilizing human induced pluripotent stem cell as an intermediate.

8 Nov 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

'Switching on' Sight using Gene Therapy

AZoLifeSciences speaks to Dr. Raymond Wong about his latest research into eye health and we could potentially 'switch on' sight using gene therapy.

20 Oct 2021

Study uncovers 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

Pioneering gene therapy has long-lasting effects on 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. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.

18 Oct 2021

Researchers identify the mechanism leading to deafness in 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.

6 Oct 2021

Gene therapy turns glial brain cells into neurons, restores visual function

A team of scientists identified a means to employ gene therapy to convert glial brain cells into neurons, reinstating visual function.

4 Oct 2021

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

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

Exploring cell and gene based therapies for Parkinson’s disease

After several decades of pre-clinical development, cell- and gene-based therapies for Parkinson's disease (PD) are now actively being explored. In this special supplement to the Journal of Parkinson's Disease on "Repairing the Parkinsonian Brain," experts highlight some of the current strategies being pursued to restore lost function and replace what is lost in the PD brain, with special emphasis on the challenges associated with translating advanced therapeutic approaches into pioneering clinical trials.

24 Sep 2021

Researchers develop novel cell reprogramming strategy to slow down glioblastomas

Gliomas are the most common primary brain tumors in adults. Among them, high-grade glioblastomas (GBMs) are particularly known to be notoriously aggressive and invasive, which makes it challenging to treat them.

16 Sep 2021

Study explores the efficiency of CasMINI, an engineered CRISPR-Cas system

A compact CRISPR-Cas system, known as CasMINI, can be employed for gene-therapy and cell-engineering applications as it is easier to deliver into cells.

7 Sep 2021

Miniature CRISPR-Cas system expands applicability and implications further

For the first time, Stanford researchers have miniaturized CRISPR for mammalian gene editing, which magnifies the scope of CRISPR technology.

3 Sep 2021

New study could help develop novel treatments for neurodegenerative diseases

Researchers have introduced a novel gene therapy program that could set the stage for new treatments for neurodegenerative diseases.

2 Aug 2021

Modification in some RNA molecules could protect against non-alcoholic fatty liver

RNA molecules while carrying genetic instructions from DNA to the protein-making machinery of cells could help guard against non-alcoholic fatty liver.

21 Jul 2021

Gene therapy may be safe and effective in treating AADC deficiency

A new study published in Nature Communications suggests that gene therapy delivered into the brain may be safe and effective in treating aromatic L-amino acid decarboxylase (AADC) deficiency.

19 Jul 2021

Novel gene therapy offers hope to children with genetic, neurodegenerative diseases

Newly devised gene therapy can help children born with AADC, a rare genetic disorder that causes developmental and physical disabilities.

13 Jul 2021

Children with rare genetic disorder experience dramatic improvements in a gene therapy trial

Children with a devastating genetic disorder characterized by severe motor disability and developmental delay have experienced sometimes dramatic improvements in a gene therapy trial launched at UCSF Benioff Children's Hospitals.

13 Jul 2021

Novel platform may lead to more improved gene therapies

Researchers from RCSI University of Medicine and Health Sciences have developed polypeptide-based materials that serve as effective vectors in delivering gene therapies.

11 Jun 2021