Abstract Chimeric Antigen Receptor (CAR) T-cells represent a breakthrough in personalized
cancer therapy. In this strategy, synthetic receptors comprised of antigen recognition …

With sweeping advances in precision delivery systems and manipulation of the genomes
and transcriptomes of various cell types, medical biotechnology offers unprecedented …

Hematopoietic stem-cell gene therapy has proven to be an effective treatment for several
primary immunodeficiencies, and yet companies in this space are withdrawing from the EU …

Friedreich's ataxia (FRDA) is an inherited, multisystemic disorder predominantly caused by
GAA hyper expansion in intron 1 of frataxin (FXN) gene. This expansion mutation …

The development of CRISPR-derived genome editing technologies has enabled the precise
manipulation of DNA sequences within the human genome. In this review, we discuss the …

Recently, clustered regularly interspaced palindromic repeats (CRISPR)-Cas9 derived
editing tools had significantly improved our ability to make desired changes in the genome …

Adenine base editors (ABE) are genome-editing tools that have been harnessed to
introduce precise A• T to G• C conversion. However, the low activity of ABE at certain sites …

Delivery of RNA using nanomaterials has emerged as a new modality to expand therapeutic
applications in biomedical research. However, the delivery of RNA presents unique …

As most patients with sickle cell disease (SCD) do not have access to curative therapies, the
availability of drug therapies that can modify disease severity remains highly desirable …

Sickle cell disease (SCD) is a common, severe genetic blood disorder. Current
pharmacotherapies are partially effective and allogeneic hematopoietic stem cell …