gene editing

The conversion of a gene-edited cell compartment is rate-limited by host cell turnover, not by the engineered graft

This deposit contains the complete computational model and analysis code for a within-host study of autologous CCR5 gene-edited HIV cure. The work shows that the conversion of a patient's CD4 T cell pool to an HIV-resistant state is rate-limited by the turnover of the patient's own unedited cells rather than by any pro...

[Corrigendum] Integrative transcriptomic analysis unveils FXR as a key regulator of intestinal stemness and inflammation in ulcerative colitis.

Following the publication of the above article, an interested reader raised several comments on the study that required clarification on the part of the authors in order to confirm the reporting of certain of the results, and to make the paper fully comprehensible. First, in the Materials and methods section, the singl...

[Corrigendum] The interaction between miR‑148a and DNMT1 suppresses cell migration and invasion by reactivating tumor suppressor genes in pancreatic cancer.

The authors are grateful to the Editor of Oncology Reports for granting them this opportunity to publish a Corrigendum, and apologize to both the Editor and the readership for any inconvenience caused.

Dynamic reprogramming and A-to-I RNA editing in neutrophils in response to tumor induction.

Results indicate a possible link between transcriptional remodeling, RNA editing, and functional polarization of HCC-induced neutrophils, and provide an exploratory framework for investigating ADAR-regulated RNA editing in tumor-immune communication.

Challenges and Strategies of Non-Coding RNA Therapies in Addressing Tumor Heterogeneity in Head and Neck Cancers.

This article systematically reviews the current research landscape of different ncRNAs in HNC at the molecular level and explores the challenges posed by heterogeneity in ncRNA therapy concerning delivery, target selection, and microenvironmental interference.

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

It is observed that in certain Streptomyces spp.

CRISPR therapy for dyslipidemia.

The potential for gene editing and epigenetic therapy for the management of dyslipidemia appears to be well tolerated, and a number of agents with the potential for robust and durable changes to atherosclerotic lipid and lipoprotein risk factors are developed.

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