CRISPR-based precision medicine for hematologic disorders: Advancements, challenges, and prospects

S Sahu, M Poplawska, SH Lim, D Dutta - Life Sciences, 2023 - Elsevier
The development of programmable nucleases to introduce defined alterations in genomic
sequences has been a powerful tool for precision medicine. While several nucleases such …

Gene Editing by Ferrying of CRISPR/Cas Ribonucleoprotein Complexes in Enveloped Virus-Derived Particles

JH Janns, JG Mikkelsen - Human Gene Therapy, 2024 - liebertpub.com
The invention of next-generation CRISPR/Cas gene editing tools, like base and prime
editing, for correction of gene variants causing disease, has created hope for in vivo use in …

Delivery of Prime editing in human stem cells using pseudoviral NanoScribes particles

T Halegua, V Risson, J Carras, M Rouyer… - Nature …, 2025 - nature.com
Prime Editing can rewrite genes in living cells by allowing point mutations, deletions, or
insertion of small DNA sequences with high precision. However, its safe and efficient …

Highly efficient CRISPR-mediated genome editing through microfluidic droplet cell mechanoporation

YJ Kim, D Yun, JK Lee, C Jung, AJ Chung - Nature communications, 2024 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)-based editing tools
have transformed the landscape of genome editing. However, the absence of a robust and …

Post-transplant G-CSF impedes engraftment of gene-edited human hematopoietic stem cells by exacerbating p53-mediated DNA damage response

D Araki, V Chen, N Redekar, C Salisbury-Ruf, Y Luo… - Cell Stem Cell, 2025 - cell.com
Granulocyte-colony-stimulating factor (G-CSF) is commonly used to accelerate recovery
from neutropenia following chemotherapy and autologous transplantation of hematopoietic …

Post-transplant administration of G-CSF impedes engraftment of gene edited human hematopoietic stem cells by exacerbating the p53-mediated DNA damage …

D Araki, V Chen, N Redekar, C Salisbury-Ruf, Y Luo… - bioRxiv, 2023 - biorxiv.org
Granulocyte colony stimulating factor (G-CSF) is commonly used as adjunct treatment to
hasten recovery from neutropenia following chemotherapy and autologous transplantation …

[引用][C] Prime editing of RYR1 gene

B Song - 2024