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Genome editing for rare diseases
Abstract Purpose of the Review Significant numbers of patients worldwide are affected by
various rare diseases, but the effective treatment options to these individuals are limited …
various rare diseases, but the effective treatment options to these individuals are limited …
Improved CRISPR/Cas9 gene editing in primary human myoblasts using low confluency cultures on Matrigel
Background CRISPR/Cas9 is an invaluable tool for studying cell biology and the
development of molecular therapies. However, delivery of CRISPR/Cas9 components into …
development of molecular therapies. However, delivery of CRISPR/Cas9 components into …
Therapeutic Genome Editing and In Vivo Delivery
AC Ramirez-Phillips, D Liu - The AAPS Journal, 2021 - Springer
Improvements in the understanding of human genetics and its roles in disease development
and prevention have led to an increased interest in therapeutic genome editing via the use …
and prevention have led to an increased interest in therapeutic genome editing via the use …
Molecular Genetic Therapies in the Muscular Dystrophies
S Nicolau, KM Flanigan - Principles and Practice of the Muscular …, 2024 - Springer
The identification of the genes causing the most common muscular dystrophies over the past
few decades has allowed the design of new classes of targeted therapeutics. A number of …
few decades has allowed the design of new classes of targeted therapeutics. A number of …
In vivo genome engineering for the treatment of muscular dystrophies
M Kustermann, MJ Rok, RD Cohn, EA Ivakine - Current Stem Cell Reports, 2020 - Springer
Abstract Purpose of Review Muscular dystrophies (MDs) are a heterogeneous collection of
inherited disorders which cause progressive muscle loss and weakness/hypotonia. Owing to …
inherited disorders which cause progressive muscle loss and weakness/hypotonia. Owing to …