In recent years, clustered regularly interspaced short palindromic repeats (CRISPRs) and
CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat …

Duchenne muscular dystrophy (DMD) is a progressive disease caused by the loss of
function of the protein dystrophin. This protein contributes to the stabilisation of striated cells …

Antisense oligonucleotide (ASO)-mediated exon skip** can restore the open reading
frame of dystrophin transcripts for Duchenne muscular dystrophy (DMD) patients. This …

Clustered regularly interspaced short palindromic repeats and associated Cas protein
(CRISPR–Cas), a powerful genome editing tool, has revolutionized gene function …

Background The development of sequence-specific precision treatments like CRISPR gene
editing therapies for Duchenne muscular dystrophy (DMD) requires sequence humanized …

Mutations that prevent the production of proteins in the DMD gene cause Duchenne
muscular dystrophy. Most frequently, these are deletions leading to reading-frame shift. The …

Advancements in genome editing enable permanent changes of DNA sequences in a site-
specific manner, providing promising approaches for treating human genetic disorders …

Duchenne muscular dystrophy (DMD) is caused by genetic mutations in the cytoskeletal-
sarcolemmal anchor protein dystrophin. Repeated cycles of sarcolemmal tearing and repair …

Definition Gene or genome editing, often known as GE, is a technique utilized to modify,
eliminate, or substitute a mutated gene at the DNA level. It serves as a valuable tool in the …

Duchenne muscular dystrophy (DMD) is a commonly encountered genetic ailment marked
by loss-of-function mutations in the Dystrophin gene, ultimately resulting in progressive …