Gene therapy and its role in the medical field have evolved drastically in recent decades.
Studies aim to define DNA-based medicine as well as encourage innovation and the further …

Recent developments in genome editing and delivery systems have opened new
possibilities for B cell gene therapy. CRISPR-Cas9 nucleases have been used to introduce …

Monogenic neuromuscular disorders are potentially treatable through gene therapy. Using
viral vectors, a therapeutic transgene aims to restore normal levels of a protein not produced …

Dentin is a major type of hard tissue of teeth and plays essential roles for normal tooth
function. Odontoblasts are responsible for dentin formation. Mutations or deficiency in …

Recent advances in stem cell biology have accelerated the pre-clinical development of cell-
based therapies for degenerative and chronic diseases. The success of this growing area …

MicroRNAs (miRNAs) provide a unique mechanism of gene regulation and play a key role in
different pathologies ranging from metabolic diseases to cancer. Thus, they are attractive …

The relevance of autophagy in neuronal health has been extensively reported in a plethora
of conditions affecting the nervous system, such as neurodegenerative diseases, cancer …

Introduction Gene therapy is an emerging topic in medicine. The first products have already
been licensed in the European Union for the treatment of immune deficiency, spinal …

Abstract p16 Ink4a is a potent cell cycle inhibitor engaged to support cell cycle arrest during
cellular senescence. However, in tumors carrying mutations in key downstream effectors …

There is an impelling need to develop effective therapeutic strategies for patients with retinal
disorders. Gleaning from the large quantity of information gathered over the past two …