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18 Μαρ 2020 · Successful gene therapies in dogs have significantly contributed to decisions to run clinical trials for several human diseases, such as Leber’s congenital amaurosis 2—LCA2 (caused by a mutation of RPE65), X-linked retinitis pigmentosa—XLRP (caused by mutation RPGR), and achromatopsia (caused by mutation of CNGB3). Promising results were ...
- Screening for Severe Combined Immunodeficiency in Neonates
The TREC assay has not been without its challenges....
- Screening for Severe Combined Immunodeficiency in Neonates
9 Οκτ 2020 · Gene therapy and gene correction techniques have developed rapidly over the last several decades, aimed at treating genetic diseases for which current treatment options are limited or...
This review discusses the known genetics of cardiomyopathies in dogs, reviews existing gene therapy strategies and the status of their development in canines, and discusses ongoing challenges in the clinical translation of these technologies for treating heart disease.
6 Μαΐ 2022 · In this article, we demonstrate the prospects for using gene therapy in veterinary medicine. The possibility of using genetic engineering allows us to create species-specific gene drugs for animals, whilst avoiding unwanted complications and side effects.
There have been many challenges, a few successes, and much promise. In 2012, Glybera was touted as the world’s first commercial gene therapy, following its approval by the European Commission for the treatment of hereditary lipoprotein lipase deficiency (LPLD) ( 2 ).
18 Μαρ 2020 · A recent study carried out on affected dogs given gene therapy at an age of 5–6 years showed that its efficacy is positively correlated with the proportion of photoreceptors remaining at the time of the treatment.
Four well-characterized diseases in canine models are discussed as they relate to other animal model availability, novel therapeutic approach, and extrapolation to human gene therapy trials. Publication types
