First patient treated in ProQR STELLAR phase 1/2 gene therapy clinical trial for Usher Syndrome Type II

by DBGen Ocular Genomics

5 years ago
Retinal Dystrophy, Research, Gene Therapy, Usher syndrome

Usher syndrome is a genetic disorder that presents recessive inheritance and is characterized by hearing loss and retinitis pigmentosa, traits that may be total or partial.

Josep Maria Aran: 'In a healthcare environment, publishing is not enough, one must go further'

by IBEC - Institut de Bioenginyeria de Catalunya

7 years ago
immune-inflammatory processes, gene therapy

Researcher Josep M. Aran leads the "Immune-inflammatory processes and gene therapy" research group within the program "Genes, Diseases and Therapy" of the Bellvitge Biomedical Research Institute (IDIB...

Researchers at CIMA describe that XBP1 could be a key agent in avoiding the accumulation of mutations

by CIMA - Centro de Investigación Médica Aplicada

7 years ago
XBP1, regeneración, protein, hepatic regeneration, liver, gene therapy

In the most recent issue of Gastroenterology, a prestigious scientific journal in the area of gastrointestinal and liver diseases, researchers at the Center for Applied Medical Research (CIMA) of the ...

Lipid nanoparticles for gene therapy

by Universidad del País Vasco

7 years ago
gene therapy, nanotechnology, Lipid nanoparticles

The journal is taking a fresh look at the advances made in this system over the last 25 years, and has included a piece of work by the PharmaNanoGene group of the UPV/EHU-University of the Basque Coun...

IDIBELL joins the ADVANCECAT project to promote advanced therapies in Catalonia

by IBEC - Institut de Bioenginyeria de Catalunya

8 years ago
human drugs, cell therapy, gene therapy, tissue engineering

Advanced therapies are human drugs based on cells (cell therapy), genes (gene therapy) or tissues (tissue engineering). To date, such drugs are being developed for applications in cancer, AIDS and car...

Strengthening gene therapy for Huntington’s disease

by Centre for Genomic Regulation

8 years ago
Huntington’s disease, zinc fingers, gene therapy

Huntington’s disease is a genetic disorder that affects around 1 in every 10,000 people and damages nerve cells in the brain. This causes neurological symptoms affecting movement, cognition and beha...

A natural polymer obtained from crustaceans is suitable for gene therapy

by Universidad del País Vasco

8 years ago
oligochitosans, crustaceans, gene therapy, biopolymer

Researchers in the UPV/EHU's Department of Pharmacy and Food Sciences have proposed using oligochitosans, which are obtained from crustaceans, for gene therapy purposes. Mireia Agirre, a researcher in...

Exploring the application in gene therapy of new polymers designed at CiQUS

by Center for Research in Biological Chemistry and Molecular Materials

8 years ago
polymers, gene therapy

Scientists at CiQUS and the University of Birmingham Chemical developed a new and fast method for the preparation of novel DNA transporters across the lipid bilayers of the cell membrane.

New polymers designed at CiQUS explore their application in gene therapy

by Center for Research in Biological Chemistry and Molecular Materials

8 years ago
polymers, gene therapy, DNA

Scientists at the Singular Research Center Biological and Molecular Materials at the University of Santiago de Compostela (CiQUS) and the University of Birmingham Chemical developed a new and fast met...

CNIO researchers fight aplastic anaemia using a therapy designed to delay ageing

by CNIO - Centro Nacional de Investigaciones Oncológicas

8 years ago
aplastic anaemia, gene therapy, Telomeres

The treatment is based on the transport of the telomerase gene to the bone marrow cells using gene therapy, a completely new strategy in the treatment of aplastic anaemia

'New clinical trials in gene therapy show the beginning of a new form of molecular medicine'

by CIMA - Centro de Investigación Médica Aplicada

10 years ago
Gene therapy, CIMA, University of Navarra

Prof. Samulski isolated and characterized during his PhD one of the most promising virus in the field of gene therapy, the adeno-associated virus

Gene-Silencing Drugs Finally Show Promise

by MIT Technology Review

10 years ago
gene therapy

The disease starts with a feeling of increased clumsiness. Spilling a cup of coffee. Stumbling on the stairs. Having accidents that are easy to dismiss—everyone trips now and then.