The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

Clinical gene therapy has been increasingly successful owing both to an enhanced
molecular understanding of human disease and to progressively improving gene delivery …

Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1
in 5000 males. Affected individuals become wheelchair bound by the age of twelve and …

In a range of human trials, viral vectors have emerged as safe and effective delivery vehicles
for clinical gene therapy, particularly for monogenic recessive disorders, but there has also …

The death of photoreceptor cells caused by retinal degenerative diseases often results in a
complete loss of retinal responses to light. We explore the feasibility of converting inner …

Gene editing-based therapeutic strategies grant the power to override cell machinery and
alter faulty genes contributing to disease development like cancer. Nowadays, the principal …

Large bone defects are usually managed by replacing lost bone with non-biological
prostheses or with bone grafts that come from the patient or a donor. Bone tissue …

Abstract COVID-19, caused by SARS-CoV-2 infection, has been prevalent worldwide for
almost a year. In early 2000, there was an outbreak of SARS-CoV, and in early 2010, a …

Gene delivery vectors based on adeno-associated viruses (AAV) have exhibited promise in
both preclinical disease models and human clinical trials for numerous disease targets …