Translational Research in Serious Human Diseases

0536561 - ÚŽFG 2021 RIV CZ eng M - Monography Chapter
Ellederová, Zdeňka - Rohiwal, Sonali Suresh
Gene therapy of monogenic diseases.
Translational Research in Serious Human Diseases. Praha: Academia, 2020 - (Kello, M.; Strnadel, J.; Klempir, J.; Roth, J.; Myslivcová-Fučíková, A.; Hansíková, H.; Kozák, I.), s. 148-153. ISBN 978-80-200-3158-7
R&D Projects: GA MŠMT(CZ) LO1609
Institutional support: RVO:67985904
Keywords : RNA interference * antisense oligonucleotides * therapy by translational read-through-inducing drugs
OECD category: Genetics and heredity (medical genetics to be 3)

Gene therapy repairs or modifies the expression of individual gene in targeted cells by transfer of nucleic material to the cells. The development of the gene therapy has been impressive over the last decade. It can reduce the production of mutated proteins causing the disease, elevate the production of proteins curing the disease, or produce modified or new proteins. Here we briefly review the main alternatives of gene targeting together with the options of delivery to the cells.
Permanent Link: http://hdl.handle.net/11104/0314329