Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

CRISPR-Cas technology has revolutionized biological research and holds great therapeutic
potential. Here, we review CRISPR-Cas systems and their latest developments with an …

Spinal muscular atrophy (SMA), the leading genetic cause of infant mortality, arises from
survival motor neuron (SMN) protein insufficiency resulting from SMN1 loss. Approved …

Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …

Spinal muscular atrophy (SMA) is caused by mutations in SMN1. SMN2 is a paralogous
gene with a C• G-to-T• A transition in exon 7, which causes this exon to be skipped in most …

Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising
innovative technology for genomic editing that offers scientists the chance to edit DNA …

In the field of medicine, it is axiomatic that the need of a precise gene-editing tool is critical to
employ therapeutic approaches toward pathogenic mutations, occurring in human genome …

Spinal muscular atrophy (SMA) is an autosomal recessive disorder with an incidence of
1/6,000–1/10,000 and is the leading fatal disease among infants. Previously, there was no …

Duchenne muscular dystrophy (DMD) is the most prevalent herediatry disease in men,
characterized by dystrophin deficiency, progressive muscle wasting, cardiac insufficiency …

During the last decades, hiPSC-derived organoids have been extensively studied and used
as in vitro models for several applications among which research studies. They can be …