Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
EA Taha, J Lee, A Hotta - Journal of Controlled Release, 2022 - Elsevier
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …
genome editing technology opened the door to provide a versatile approach for treating …
Engineering a precise adenine base editor with minimal bystander editing
Adenine base editors (ABEs) catalyze A-to-G transitions showing broad applications, but
their bystander mutations and off-target editing effects raise safety concerns. Through …
their bystander mutations and off-target editing effects raise safety concerns. Through …
Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors
The viral delivery of base editors has been complicated by their size and by the limited
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
packaging capacity of adeno-associated viruses (AAVs). Typically, dual-AAV approaches …
Precise genomic editing of pathogenic mutations in RBM20 rescues dilated cardiomyopathy
Mutations in RNA binding motif protein 20 (RBM20) are a common cause of familial dilated
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …
cardiomyopathy (DCM). Many RBM20 mutations cluster within an arginine/serine-rich (RS …
Therapeutic approaches for Duchenne muscular dystrophy
TC Roberts, MJA Wood, KE Davies - Nature Reviews Drug Discovery, 2023 - nature.com
Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
priority candidate for molecular and cellular therapeutics. Although rare, it is the most …
Base-editing mutagenesis maps alleles to tune human T cell functions
CRISPR-enabled screening is a powerful tool for the discovery of genes that control T cell
function and has nominated candidate targets for immunotherapies,,,,–. However, new …
function and has nominated candidate targets for immunotherapies,,,,–. However, new …
Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice
Y Zhou, C Zhang, W Xiao, RW Herzog… - Nature Communications, 2024 - nature.com
Current gene therapy for Duchenne muscular dystrophy (DMD) utilizes adeno-associated
virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for …
virus (AAV) to deliver micro-dystrophin (µDys), which does not provide full protection for …
CRISPR modeling and correction of cardiovascular disease
Cardiovascular disease remains the leading cause of morbidity and mortality in the
developed world. In recent decades, extraordinary effort has been devoted to defining the …
developed world. In recent decades, extraordinary effort has been devoted to defining the …
CRISPR/Cas systems: Delivery and application in gene therapy
The CRISPR/Cas systems in prokaryotes such as bacteria and archaea are the adaptive
immune system to prevent infection from viruses, phages, or other foreign substances. When …
immune system to prevent infection from viruses, phages, or other foreign substances. When …