The replication-defective, non-pathogenic, nearly ubiquitous single-stranded adeno-
associated viruses (AAVs) have gained importance since their discovery about 50 years …

Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic
option for treating genetic diseases and some cancers. However, there are challenges in …

Advances in genome sequencing have greatly facilitated the identification of genomic
variants underlying rare neurodevelopmental and neurodegenerative disorders …

In this communication, the authors offer considerations for how the scientific community can
capitalize on decades of translational science advances and experiential knowledge to …

The promise of viral vector‐based gene therapy (GT) as a transformative paradigm for
treating severely debilitating and life‐threatening diseases is slowly coming to fruition with …

Duchenne muscular dystrophy (DMD) is a fatal genetic disease affecting children that is
caused by a mutation in the gene encoding for dystrophin. In the absence of functional …

The literature thoroughly describes the challenges of pediatric drug development for rare
diseases. This includes (1) generating interest from sponsors,(2) small numbers of children …

Highlights•CRISPR-based genome editing tools offer promise for treating genetic
disease.•In vivo CRISPR therapeutics require potent and safe delivery vectors.•Biological …

Abstract Background Approximately 4–8% of the world suffers from a rare disease. Rare
diseases are often difficult to diagnose, and many do not have approved therapies. Genetic …

Orphan drug designation (ODD) is an important program intended to facilitate the
development of orphan drugs in the United States. An orphan drug benefiting pediatric …