Gene therapy in paediatrics is an innovative treatment approach
for addressing various genetic disorders that present in childhood. Gene and
cell therapies have been developed and approved for a growing number of
pediatric diseases, with ongoing research for additional innovative treatment
options for the future. Each therapy is tailored to the specific disease and
targets a specific genetic alteration or cell population. The development of
new therapies is a complex and regulated process, with strict oversight at
regional and European levels. Gene therapy has three facets such as, gene
silencing using siRNA, shRNA and miRNA; gene addition introducing a functional
gene that contains instructions for the cell to produce a specific protein; and
finally gene editing based therapy where mutations are modified using specific
nucleases such as zinc-finger nucleases (ZFNs), transcription activator-like
effector nucleases (TALENs) and clustered regulatory interspaced short tandem
repeats (CRISPR)/CRISPR-associated protein (Cas)-associated nucleases. This
review explores the emerging gene and cell therapy strategies for rare
pediatric genetic disorders. The success of gene therapy in treating genetic
diseases depends on understanding the specific characteristics and function of
the relevant gene, the genetic changes that cause disease, and the regulatory
systems that affect gene expression. Rare pediatric neurogenetic diseases
typically manifest early in life, lack specific treatment options, have high
mortality rates, and present a significant threat to children's health and
survival. A one-time genetic approach is desirable in many rare pediatric
diseases and has been established only in a few pediatric diseases to date.
Rare genetic diseases in childhood are an important factor as a healthcare concern
due to high costs for the healthcare system, especially in gene therapy.
Author(s)
Details
Stefan Bittmann
Department of Pediatrics, Ped Mind Institute, Hindenburgring 4,
D-48599 Gronau, Germany and Shangluo Vocational and Technical College, Shangluo,
726000, Shaanxi, China.
Please see the book here:- https://doi.org/10.9734/bpi/mbrao/v4/5688
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