The bacteria-derived CRISPR/Cas (an acronym for regularly interspaced short palindromic
repeats/CRISPR-associated protein) system is currently the most widely used, versatile, and …

The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases
holds tremendous potential. This innovative approach combines the advantages of …

Genodermatoses represent a large group of inherited skin disorders encompassing
clinically-heterogeneous conditions that manifest in the skin and other organs. Depending …

Synthetic biology (synbio) tools, such as chimeric antigen receptors (CARs), have been
designed to target, activate, and improve immune cell responses to tumors. These therapies …

Recent innovations in the field of gene therapy have paved the way for advances towards
developing genome editing medicines. Despite these steps forward, challenges with viral …

Acute Lymphoblastic Leukemia (ALL) is the predominant hematological malignancy in
pediatric populations, originating from B-or T-cell precursors within the bone marrow. The …

An expanded CAG repeat in the huntingtin gene (HTT) causes Huntington's disease (HD).
Since the length of uninterrupted CAG repeat, not polyglutamine, determines the age-at …

Hepatitis B virus (HBV) has afflicted humankind for decades and there is still no treatment
that can clear the infection. The development of recombinant adeno-associated virus (rAAV) …

In utero gene editing (IUGE) is a potential treatment for inherited diseases that cause
pathology before or soon after birth. Preexisting immunity to adeno-associated virus (AAV) …

Adoptive T cell transfer has shown great success in treating blood cancers, resulting in a
growing number of FDA-approved therapies using chimeric antigen receptor (CAR) …