type your search, and then press enter

Noticias y Eventos

Descubra las novedades en el campo de las patologías oculares hereditarias

Blog operado por el equipo de DBGen que actualiza terapias, noticias y eventos relacionados con las enfermedades hereditarias oculares. Le invitamos a participar en este foro.

TwinPE: An improved genome-editing tool that allows inserting entire genes for gene therapy

Researchers at the Broad Institute of Harvard University have just reported in the scientific journal Nature Biotechnology an improved version of prime editing, the highly accurate genome editing method developed in 2019 by the same team. This new version, called twin prime editing (twinPE), allows insertion of DNA fragments of the size of a gene into specific sites of the...

read more

Innovation in gene therapy designs for retinal diseases

17/12/21 - Therapy, Gene Therapy,

Gene therapy aims to recover the normal function of the cells of a patient to obtain a therapeutic benefit by introducing genetic material. The benefit being, for example, in improving the immune system response to fight cancer or correcting a genetic disorder by rescuing gene function. Scientists harness the natural ability of viruses to transfer their genetic material to infected...

read more

Innovative gene therapy for autosomal dominant retinitis pigmentosa

Mutations in the RHO gene are the main cause of dominant forms of retinitis pigmentosa (RP). RHO dictates the production of rhodopsin, a transmembrane protein present in large quantities in the outer segment of rod photoreceptor cells which, together with 11-cis-retinal, initiates the phototransduction cascade. Mutations in RHO often cause photoreceptor toxicity because they negatively affect the function of the...

read more

Novel high-precision gene therapy to correct an eye disease in mice

Researchers from the Seoul National University (Korea) have applied for the first an improved genome-editing methodology, named prime editing to correct the genetic defects of two animal models of human disease: a liver disease (hereditary tyrosinemia) and an eye disease (Leber congenital amaurosis). This high-precision genome-editing tool, developed only two years ago by the team of David R. Liu from...

read more

Encouraging advances in therapies for Leber congenital amaurosis

Editas Medicine CRISPR therapy for CEP290 gene Editas Medicine, a company specialising in the development of gene editing therapies, has announced the recruitment of paediatric patients to the Brilliance trial. This phase 1/2 clinical trial began last year and is based on the CRISPR gene editing methodology to excise the intron mutation c.2991+1655A>G, which produces an aberrant messenger RNAs (see...

read more

LHON gene therapy affords sustained improvement in visual acuity paving the way to approval

17/03/21 - Gene Therapy,

The biopharma company GenSight Biologics, specialized in developing gene therapies for retinal degenerative diseases, recently published results of two Phase III clinical trials on the effect of LUMEVOQ gene therapy for Leber Hereditary Optic Neuropathy (LHON) patients. In September 2020, GenSight requested approval of LUMEVOQ to the European Medicines Agency (EMA) to treat LHON patients carrying a mutation in the...

read more
1 2 3 5
Copyright © 2022. All Rights Reserved -