Genetic therapies for cystic fibrosis lung disease
Sign Up to like & getrecommendations! Published in 2017 at "Current Opinion in Pharmacology"
DOI: 10.1016/j.coph.2017.10.006
Abstract: HighlightsGene editing strategies for gene therapy of cystic fibrosis.Chemically modified mRNA for CFTR protein replacement therapy.Better viral and non‐viral vectors targeted to lung epithelium.Human and animal models for CF. &NA; Gene therapy for cystic fibrosis… read more here.
Keywords: non viral; cystic fibrosis; fibrosis; gene therapy ... See more keywords
Genetic Therapies for Hearing Loss: Accomplishments and Remaining Challenges
Sign Up to like & getrecommendations! Published in 2019 at "Neuroscience Letters"
DOI: 10.1016/j.neulet.2019.134527
Abstract: More than 15 years have passed since the official completion of the Human Genome Project. Predominantly due to this project, over one hundred genes have now been linked to hearing loss. Although major advancements have… read more here.
Keywords: hearing loss; therapies hearing; gene therapy; genetic therapies ... See more keywords
Genetic Therapies for Sickle Cell Disease.
Sign Up to like & getrecommendations! Published in 2018 at "Pediatric clinics of North America"
DOI: 10.1016/j.pcl.2018.01.008
Abstract: Sickle cell disease is the most prevalent monogenic disorder worldwide and curative therapies are limited to hematopoietic stem cell transplant to the few with matched donors. Gene therapy has curative potential, whereby autologous hematopoietic stem… read more here.
Keywords: genetic therapies; cell disease; cell; therapies sickle ... See more keywords
Genetic therapies for sickle cell disease.
Sign Up to like & getrecommendations! Published in 2018 at "Seminars in hematology"
DOI: 10.1053/j.seminhematol.2018.04.014
Abstract: After decades with few novel therapeutic options for sickle cell disease (SCD), autologous hematopoietic stem cell (HSC) based genetic therapies including lentiviral gene therapy (GT), and genome editing (GE) now appear imminent. Lentiviral GT has… read more here.
Keywords: genetic therapies; cell disease; cell; therapies sickle ... See more keywords
Benefits and Shortcomings of Laboratory Model Systems in the Development of Genetic Therapies.
Sign Up to like & getrecommendations! Published in 2022 at "Klinische Monatsblatter fur Augenheilkunde"
DOI: 10.1055/a-1757-9879
Abstract: Gene therapeutic approaches promise treatment or even a cure of diseases that were previously untreatable. Retinal gene therapies tested in clinical trials comprise a wide range of different strategies, including gene supplementation therapies, in vivo… read more here.
Keywords: genetic therapies; development genetic; benefits shortcomings; gene ... See more keywords
Biological and genetic therapies for the treatment of Duchenne muscular dystrophy
Sign Up to like & getrecommendations! Published in 2022 at "Expert Opinion on Biological Therapy"
DOI: 10.1080/14712598.2022.2150543
Abstract: ABSTRACT Introduction Duchenne muscular dystrophy is a lethal genetic disease which currently has no cure, and poor standard treatment options largely focused on symptom relief. The development of multiple biological and genetic therapies is underway… read more here.
Keywords: duchenne muscular; therapy; biological genetic; muscular dystrophy ... See more keywords
Are Genetic Therapies for Epilepsy Ready for the Clinic?
Sign Up to like & getrecommendations! Published in 2023 at "Epilepsy Currents"
DOI: 10.1177/15357597231176234
Abstract: In recent years, there has been a significant increase in preclinical studies to test genetic therapies for epilepsy. Some of these therapies have advanced to clinical trials and are being tested in patients with monogenetic… read more here.
Keywords: epilepsy ready; ready clinic; genetic therapies; therapies epilepsy ... See more keywords