Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research

Mutation-induced phenotypic plasticity drives formation of a pro-metastatic tumour-intrinsic niche

It is argued that formation of a tumour-intrinsic niche is a prerequisite for BRAF-mutant CRC seeding to distant organs and that interference with niche formation may help avoid metastatic relapse.

Unlocking non-model organisms with CRISPR-Cas: A roadmap for sustainable biotechnology.

It is concluded that bridging the gap between foundational CRISPR research and its real-world applications is imperative and future efforts should focus on democratizing tools via open-source platforms, advancing delivery systems, and fostering sustainable innovation through synthetic biology integration to fully realize the transformative potential of genome editing in organisms beyond model organisms.

Erratum: Resolution of hepatic fibrosis after ZFN-mediated gene editing in the PiZ mouse model of human α1-antitrypsin deficiency.

Virus-like particles enable targeted gene engineering and pooled CRISPR screening in primary human myeloid cells.

A virus-like particle (VLP)-based toolkit that delivers diverse CRISPR editing modalities to human monocytes, macrophages and dendritic cells with high efficiency while preserving viability and innate immune responsiveness is presented.

CRISPR/Cas9-Mediated Disruption of Duplicated Sizzled Genes Induces Twin-Tail-like Caudal Bifurcation in Goldfish (Carassius auratus)

Findings provide direct functional evidence that szl regulates median caudal patterning in goldfish and suggest that szl-dependent modulation of the Chordin/BMP network can generate twin-tail-like caudal morphology.

Recent applications of CRISPR-Cas systems in animal models of human cardiovascular disease: Review article

Cardiovascular disease (CVD) causes more deaths, more years of life lost, and more years lived with disability than any other major category of disease worldwide. Gene editing technologies, including the CRISPR-Cas9 system and its offshoots, are increasingly applied in CVD animal research models to identify genetic drivers of disease processes and to develop and test targeted therapeutics. To explore the potential impact and limitations of using animal models for these applications, this review examines studies in which the creation of a CRISPR-Cas9 animal model has been used to find genetic drivers and develop therapies. Studies were identified using OVID Medline, searching the past 10 years of the primary literature across 4 CVD domains: congenital heart disease, hypertrophic cardiomyopathy, heart failure, and atherosclerosis. The studies illustrate the vast range of gene editing applications and the value of models that parallel human genetic pathophysiology for specific disease processes. Besides furthering anatomical, clinical, and natural historical understanding of CVD pathologies and providing biological substrates in the development of screening and diagnostic tools, animal models support foundational stages in the development of genetic therapeutics, from gene target identification to discovery and development of gene-editing therapy mechanisms and delivery vehicles, through conducting therapeutic trials to assess the risk-benefit ratio.