gene editing

In vivo HSPC gene therapy of hemoglobinopathies without drug selection of corrected cells

In vitro studies in an erythroid progenitor cell line and primary CD34+ cells demonstrated that the EPORW439* variant conferred a strong proliferative advantage to therapeutically modified erythroid progenitors, establishing a strategy to amplify the therapeutic benefit of otherwise limited in vivo HSPC gene editing fo...

Assembly and structural analysis of the mitochondrial genome of six Cucumis species

Plant mitochondrial genomes exhibit remarkable structural plasticity and evolutionary complexity; however, their diversity and evolutionary mechanisms remain poorly understood in many crop lineages. In this study, we systematically characterized the mitochondrial genome structures of Cucumis species to provide new insi...

A novel minimally humanized mouse model of aniridia for preclinical evaluation of CRISPR gene editing strategies

A minimally humanized mouse model of aniridia, carrying a FLAG-tagged Pax6 and the most common recurring pathogenic aniridia-patient variant, introduced for the first time in mice is generated and tested, supporting successful generation and phenotypic validation of the humanized mouse.

CRISPR‐based therapeutic and modelling approaches in Huntington's disease: Progress, challenges and future directions

No approach yet combines adequate central nervous system distribution, mutant‐allele selectivity, durable neurological benefit, and established long‐term safety in HD research and development, but no approach yet combines adequate central nervous system distribution, mutant‐allele selectivity, durable neurological bene...

Strain-specific outcomes of cytosine-base editing in Streptomyces

ABSTRACT The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system has facilitated gene editing of different organisms. Specifically, programmable base editing enables stable conversion of a single nucleotide to another nucleotide without causing DNA double-strand breaks. Cas9-derived base edito...

CRISPR therapy for dyslipidemia.

PURPOSE OF REVIEW Several lipid and lipoprotein risk factors play an important role in the causality of atherosclerotic cardiovascular disease. Many therapies are limited by suboptimal long-term adherence. This narrative review summarizes the potential for gene editing and epigenetic therapy for the management of dysli...

Gene-editing technologies and gene delivery modalities for neurodegenerative disorders: Current advances and challenges

Establishment of an efficient in-vitro regeneration system from leaves of Phalaenopsis hybrida cv. ‘Big Chili’

GENESIS-SHIELD: an interpretable ensemble for anomaly detection in CRISPR genomic-workflow security

The rapid advancement of CRISPR-based gene editing has introduced digital-workflow security and integrity challenges: unauthorized modifications, temporal inconsistencies, and duplicated provenance records can compromise the auditability of editing logs. These are distinct from the biological risks of editing its...

CRISPR-Based Mediated Reactivation of Fetal Hemoglobin as a Therapeutic Strategy for Hemoglobinopathies: Evidence from Preclinical to Clinical Trials in Sickle Cell Disease and β-Thalassemia.

β-Hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia, are inherited disorders caused by mutations in the β-globin gene (HBB), leading to defective production of adult hemoglobin (HbA), vaso-occlusive crises, and rapid destruction of erythrocytes as they leave the bone marrow, resulting in hemolyt...

Gene therapy in amyotrophic lateral sclerosis (ALS)

Gene therapy in limb-girdle muscular dystrophy (LGMD)