Adeno-associated virus vector as a platform for gene therapy delivery
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the
treatment of a variety of human diseases. Recent advances in developing clinically …
treatment of a variety of human diseases. Recent advances in developing clinically …
Gene editing and CRISPR in the clinic: current and future perspectives
Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
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 …
Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque
KT McCullough, SL Boye, D Fajardo, K Calabro… - Human gene …, 2019 - liebertpub.com
Mutations in GUCY2D, the gene encoding retinal guanylate cyclase-1 (retGC1), are the
leading cause of autosomal dominant cone–rod dystrophy (CORD6). Significant progress …
leading cause of autosomal dominant cone–rod dystrophy (CORD6). Significant progress …
A review on molecular scissoring with CRISPR/Cas9 genome editing technology
Genome editing is a technology to make specific changes in the DNA of a cell or an
organism. It has significantly altered the landscape of life sciences, facilitating the …
organism. It has significantly altered the landscape of life sciences, facilitating the …
In Vivo Genome Editing Partially Restores Alpha1-Antitrypsin in a Murine Model of AAT Deficiency
CRISPR (clustered regularly interspaced short palindromic repeats) genome editing holds
promise in the treatment of genetic diseases that currently lack effective long-term therapies …
promise in the treatment of genetic diseases that currently lack effective long-term therapies …
Evaluation of cytosine base editing and adenine base editing as a potential treatment for alpha-1 antitrypsin deficiency
Alpha-1 antitrypsin deficiency (AATD) is a rare autosomal codominant disease caused by
mutations within the SERPINA1 gene. The most prevalent variant in patients is PiZ …
mutations within the SERPINA1 gene. The most prevalent variant in patients is PiZ …
[PDF][PDF] mRNA and gene editing: Late breaking therapies in liver diseases
N Zabaleta, L Torella, ND Weber… - …, 2022 - Wiley Online Library
The efficient delivery of RNA molecules to restore the expression of a missing or
inadequately functioning protein in a target cell and the intentional specific modification of …
inadequately functioning protein in a target cell and the intentional specific modification of …
Delivery of genome-editing biomacromolecules for treatment of lung genetic disorders
T Wan, Y Ping - Advanced drug delivery reviews, 2021 - Elsevier
Genome-editing systems based on clustered, regularly interspaced, short palindromic
repeat (CRISPR)/associated protein (CRISPR/Cas), are emerging as a revolutionary …
repeat (CRISPR)/associated protein (CRISPR/Cas), are emerging as a revolutionary …
Clarifying the risk of lung disease in SZ alpha-1 antitrypsin deficiency
AN Franciosi, BD Hobbs, OJ McElvaney… - American Journal of …, 2020 - atsjournals.org
Rationale: The ZZ genotype of alpha-1 antitrypsin deficiency (AATD) is associated with
chronic obstructive pulmonary disease (COPD), even among never-smokers. The SZ …
chronic obstructive pulmonary disease (COPD), even among never-smokers. The SZ …