Ribonucleoproteins Mediated Efficient In Vivo Gene Editing in Skin Stem Cells.
Sign Up to like & getrecommendations! Published in 2019 at "Methods in molecular biology"
DOI: 10.1007/7651_2018_115
Abstract: The clustered regularly interspaced, short palindromic repeats (CRISPR)-Cas9 system functions like an adaptive immune system in a variety of microbes and has recently been engineered as a powerful tool for manipulating genomic sequences in a… read more here.
Keywords: skin stem; stem cells; vivo gene; gene editing ... See more keywords
Fe3+-coordinated multifunctional elastic nanoplatform for effective in vivo gene transfection.
Sign Up to like & getrecommendations! Published in 2020 at "ACS applied materials & interfaces"
DOI: 10.1021/acsami.9b19585
Abstract: The common phenomenon that the non-viral vectors have much lower transfection efficiency in vivo than in vitro greatly restricts its further developments and applications. Possible reasons are lacking targeting ability, elimination by the reticuloendothelial system… read more here.
Keywords: gene; vivo gene; pei r12; transfection ... See more keywords
Challenges and opportunities when transitioning from in vivo gene replacement to in vivo CRISPR/Cas9 therapies – a spotlight on hemophilia
Sign Up to like & getrecommendations! Published in 2022 at "Expert Opinion on Biological Therapy"
DOI: 10.1080/14712598.2022.2090241
Abstract: ABSTRACT Introduction Currently, a few in vivo gene replacement therapies are commercially available, with many in clinical development for the treatment of some inherited monogenic diseases. These disorders arise from mutations in genes encoding essential… read more here.
Keywords: gene replacement; replacement therapies; vivo gene; challenges opportunities ... See more keywords
Ex vivo gene therapy for lysosomal storage disorders: future perspectives
Sign Up to like & getrecommendations! Published in 2023 at "Expert Opinion on Biological Therapy"
DOI: 10.1080/14712598.2023.2192348
Abstract: ABSTRACT Introduction Lysosomal storage disorders (LSD) are a group of monogenic rare diseases caused by pathogenic variants in genes that encode proteins related to lysosomal function. These disorders are good candidates for gene therapy for… read more here.
Keywords: storage disorders; gene therapy; vivo gene; lysosomal storage ... See more keywords
In vivo gene therapy for canine SCID-X1 using cocal-pseudotyped lentiviral vector.
Sign Up to like & getrecommendations! Published in 2020 at "Human gene therapy"
DOI: 10.1089/hum.2020.127
Abstract: Hematopoietic stem and progenitor cell (HSPC)-based ex vivo gene therapy has demonstrated clinical success for X-linked severe combined immunodeficiency (SCID-X1) patients who lack a suitable donor for HSPC transplantation. Nevertheless, this form of treatment is… read more here.
Keywords: vector; gene therapy; scid; vivo gene ... See more keywords
DOTAP-Incorporated PEG-PLGA Nanoparticles for Efficient In Vitro and In Vivo Gene Delivery.
Sign Up to like & getrecommendations! Published in 2018 at "Journal of biomedical nanotechnology"
DOI: 10.1166/jbn.2018.2470
Abstract: The application of nucleic acids as therapeutic agents has been obstructed by the lack of efficient and safe systems for the delivery of the therapeutic DNA into target cells. Nanoparticle (NP) vectors based on biocompatible… read more here.
Keywords: delivery; gene delivery; vivo gene; peg plga ... See more keywords
In vivo Gene Therapy to the Liver and Nervous System: Promises and Challenges
Sign Up to like & getrecommendations! Published in 2021 at "Frontiers in Medicine"
DOI: 10.3389/fmed.2021.774618
Abstract: In vivo genetic engineering has recently shown remarkable potential as a novel effective treatment for an ever-growing number of diseases, as also witnessed by the recent marketing authorization of several in vivo gene therapy products.… read more here.
Keywords: gene therapy; vivo gene; gene; nervous system ... See more keywords
In Vivo and Ex Vivo Gene Electrotransfer in Ophthalmological Disorders
Sign Up to like & getrecommendations! Published in 2022 at "Biomedicines"
DOI: 10.3390/biomedicines10081889
Abstract: The aim of this document is to present an overview of gene electrotransfer in ophthalmological disorders. In order to ensure an adequate variety of the assessed studies, several electronic databases were considered and studies published… read more here.
Keywords: ophthalmological disorders; gene electrotransfer; vivo gene; vivo vivo ... See more keywords