That by using several model systems including mouse, zebrafish

That the base editors can be
used for correction of single base genetic defects has been demonstrated by
using several model systems including mouse, zebrafish and Xenomus. Efficiency and effectiveness of these base editors has
also been demonstrated using human cell lines and zygotes/embryos. For
instance, in a seminal recent study, using mouse/human cells, it was shown that the mutations (C®Tor G®A) associated with Alzheimer’s (APOE4, Cys158Arg mutation: potent Alzheimer’s risk
factor), and some
cancers (p53 Tyr163Cys mutation, associated with cancer) can be treated through
base editing15.  Standard
CRISPR­Cas9 method of genome editing may not help in many of these cases, but a
safe and reliable delivery of base editors (BEs) in the form of molecular machine can be
successfully used with no side effects. In this connection, concerns if any,
are being addressed by researchers around the world, so that in the near future
gen therapy based on base-editors will become available, although it may take
time for them to reach the doctors clinics.

      Several reports are available, where
corrections of individual genes has been demonstrated even at the organismal
levels in model systems like mouse, zebrafish and Xenopus, where altered embryos
were transplanted in pesudopegnant surrogate mothers and mutant offspring
obtained26,27. The gene tyr
encoding tyrosine pigment causing albinism has been successfully used in
several of these studies. In China, a single
base mutation for a blood disorder was corrected in human embryos using a
molecular machine in the form of base editor, although the embryos were not
allowed to develop further.

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