Long-term efficacy of AAV5-based gene therapy   to treat day blind sheep with achromatopsia

(Mary Ann Liebert, Inc./Genetic Engineering News) A study of a large animal model of achromatopsia caused by a mutation in the CNGA3 gene that was treated with a single injection of CNGA3 gene therapy delivered using an AAV5 vector revealed findings reported long-term follow-up findings that show promise for the efficacy and safety of this therapeutic approach.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news

Related Links:

Abstract Rapid progress in knowledge of the organization of the dog genome has facilitated the identification of the mutations responsible for numerous monogenic diseases, which usually present a breed-specific distribution. The majority of these diseases have clinical and molecular counterparts in humans. The affected dogs have thus become valuable models for preclinical studies of gene therapy for problems such as eye diseases, immunodeficiency, lysosomal storage diseases, hemophilia, and muscular dystrophy. Successful gene therapies in dogs have significantly contributed to decisions to run clinical trials for ...
Source: J Appl Genet - Category: Genetics & Stem Cells Authors: Tags: J Appl Genet Source Type: research
RARITAN, NJ, March 2, 2020 – The Janssen Pharmaceutical Companies of Johnson &Johnson announced today that the European Medicines Agency (EMA) has granted both PRIME (PRIority MEdicines) and Advanced Therapy Medicinal Product (ATMP) designations to the company’s adeno-associated virus (AAV)-RPGR gene therapy product for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP). PRIME is awarded to increase interactions, optimize development plans and accelerate innovative treatments where there is unmet medical need. Similarly, ATMP status is granted to medicines that are based on gene...
Source: Johnson and Johnson - Category: Pharmaceuticals Tags: Innovation Source Type: news
ConclusionsLow vision and nystagmus observed by parents, particularly in the first year of life, may represent a red flag, prompting an appropriate ophthalmological workup for inherited retinal dystrophy.
Source: Italian Journal of Pediatrics - Category: Pediatrics Source Type: research
What if you could have a heat-map vision to search through an abandoned terrain? What if you could filter out unpleasant odors of urban decay every now and then? Can you imagine hearing ultrasounds better than bats? And what about lifting 20 tons or having a third, bionic arm? With the rapid advancement of technologies, the future of healthcare might not just be about being healthy, but even augmenting our bodies and “upgrading” ourselves. Where would you draw the line between being a human and a cyborg? Superhuman traits in sight: perceiving colors as sounds The wish for a perfect human body with enhance...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Biotechnology Cyborgization Health Sensors & Trackers artificial brain-computer interface digital tattoo exoskeleton future Healthcare implant Innovation Medicine Source Type: blogs
Condition:   Achromatopsia Intervention:   Biological: AAV- CNGA3 Sponsor:   MeiraGTx UK II Ltd Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
s S Abstract Gene therapy holds promise for treating previously untreatable retinal disorders. The most promising approaches use gene transfer vectors derived from adeno-associated virus (AAV) to supplement a gene function in the affected cell type. One example is gene therapy for achromatopsia which affects daylight vision. In this case, recombinant AAV (rAAV) vectors are being developed to specifically target cone photoreceptors. Development of rAAV vectors could be facilitated by the use of in vitro models. In this chapter we provide a protocol which utilizes mouse 661W cells, an in vitro model of cone photorec...
Source: Mol Biol Cell - Category: Molecular Biology Authors: Tags: Methods Mol Biol Source Type: research
Hug shirts, smellphones, virtual tastes, bionic eyes and hearing aids doing translations – just a few keywords showing how technology will take human perception to a higher level in the future. Innovative healthcare solutions will go way beyond improving our senses when we experience problems, they will augment our capabilities and open new horizons for humanity. Let’s jump into the pool of details. How humans perceive the sensory cacophony called the world Car. Flower. Smartphone. Leaf. Shadow. Ponytail. Red Sweater. Monitor. Water. Coffee. Beeps. Sidney Bechet tunes. Bicycle. Laugh. Light breeze. Holiday mem...
Source: The Medical Futurist - Category: Information Technology Authors: Tags: Biotechnology Cyborgization Health Sensors & Trackers Medical Professionals Patients body augmentation future Healthcare hearing human human perception Medicine sense sensing smell taste touch vision Source Type: blogs
More News: Achromatopsia (ACHM) | Eyes | Gene Therapy | Genetics | Study