Recent advances and feasible strategies of CRISPR/Cas9-based genome editing for hemophilia therapy are outlined, the potential applications of next-generation gene editing and vector delivery technologies are highlighted, and new insights are offered for the advantages, limitations, and future directions for these nove...
Xiao-Yu Zhang, Kai-Di Xu, Shi-Hui Zou et al.· Frontiers in Genome Editing· 0 citations
In this course, we investigate into the complex landscape of gene editing within aclinical framework, focusing on the generation of a comprehensive map detailingcurrent delivery methodologies employed for in vivo gene editing across variousanimal models and clinical scenarios, with some insight for ex vivo studies. The...
Yonglun Luo, Karim Benabdellah, Mariana Köber et al.· Zenodo (CERN European Organi...· 0 citations
Hepatitis is a leading cause of morbidity and mortality. It is a significant global public health challenge, affecting more than 300 million people worldwide. Achieving a hepatitis free future requires an integrated approach that combines medical innovation, public health initiatives, and equitable healthcare delivery....
Mugdivari Sangeetha, G. Akshitha, T .Satvika, V. Srinidhi, V. Uma MaheswarRao· Zenodo (CERN European Organi...· 0 citations
This dataset contains the digitized treatments in Plazi based on the original journal article Moya, Gabriela, Yan, Zheng-Fei, Chu, Dong-Hun, Won, KyungHwa, Yang, Jung-Eun, Wang, Qi-Jun, Kook, Moo-Chang, Yi, Tae-Hoo (2018): Deinococcus hibisci sp. nov., isolated from rhizosphere of Hibiscus syriacus L. (mugunghwa flower...
Gabriela Moya, Zheng‐Fei Yan, Dong-Hun Chu et al.· Global Biodiversity Informat...· 0 citations
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Efficient multiplex genome editing is essential for improving complex traits in livestock that are governed by multiple genes. However, achieving high‐efficiency simultaneous editing at multiple loci remains challenging for cytosine base editors (CBEs), largely due to variable editing efficiencies and limited deamina...
Shao-Shuai Wang, Yu Wang, Deng-Feng Bi et al.· Animal Research and One Heal...· 0 citations
An in-depth literature review of the current approaches to informed consent for somatic gene editing and gene therapy and identify potential areas for improvement, specifically in relation to SCD in sSA is conducted.
Patrick Ohiani Moru, O. Nnodu, V. Nembaware et al.· BMC Medical Ethics· 0 citations
Tomato (Solanum lycopersicum) faces increasing pressure from both biotic threats, including bacterial, fungal, and viral pathogens, and abiotic stresses such as drought, salinity, heat, and cold, all of which substantially reduce productivity under changing climatic conditions. CRISPR/Cas-based genome editing has emerg...
Xi-Jun Sun, Ke Wang, Ming-Ming Gao et al.· Crop Breeding, Genetics and...· 0 citations
Genetic neuromuscular disorders (NMDs) that predominantly affect muscle tissue are individually rare but cumulatively are responsible for significant clinical and economic burdens on patients and healthcare systems. Few effective, targeted therapies exist outside of the Duchenne muscular dystrophy (DMD) landscape, high...
Purpose: The short lifespan of primary normal choroidal melanocytes (NCMs) in vitro represents a major barrier to mechanistic, functional, and translational studies of choroid biology and uveal melanoma (UM). This study aimed to establish and characterize immortalized human NCM lines that retain melanocytic function, m...
Aurélie Fuentes-Rodriguez, Andrew Mitchell, Vincent Gelinas et al.· Investigative Ophthalmology...· 0 citations
What's Changed ZFIN-10286: soften diff highlight + add gene-symbol columns to report by @rtaylorzfin in https://github.com/ZFIN/zfin/pull/1919 ZFIN-10373: Solr backup/restore: per-instance backup, restore from shared zfindb by @rtaylorzfin in https://github.com/ZFIN/zfin/pull/1929 ZFIN-10388: Fix manually named STRs ig...
Christian Pich, Ryan Taylor, zfinryanm et al.· Zenodo (CERN European Organi...· 0 citations
This review discusses this emerging field and its transformative potential for translational neuropharmacology, focusing on therapies for neurodevelopmental and neurodegenerative disorders.
The results showed that AAV2-OCRL demonstrated the highest transduction efficiency in patient iPSC-derived trabecular meshwork models (iHTM) among the three AAV-OCRL vectors evaluated, establishing it as a promising delivery vector.
Si-Yu Chen, Zhi-Quan Liu, Wen-Min Wang et al.· Advancement of science· 0 citations
Zero-knowledge proofs can verify computing results without rerunning workloads or exposing sensitive data. Magnetar helps bring them into the mainstream with support for standard programming languages, major speed gains, and quantum-resistant security. The post Proof-based general-purpose computing with Magnetar appeared first on Microsoft Research.