RNA-targeting and gene editing therapies for transthyretin amyloidosis

Alberto Aimo, Vincenzo Castiglione, Claudio Rapezzi et al.

Heterologous and endogenous U6 snRNA promoters enable CRISPR/Cas9 mediated genome editing in Aspergillus niger

Xiaomei Zheng, Ping Zheng, Jibin Sun

Gene Editing Successfully Corrects 2 Amino Acid Disorders: In 2 preclinical studies using CRISPR‐mediated gene editing, phenylketonuria and hereditary tyrosinemia type 1 were corrected

Trem-Cel, a CRISPR/Cas9 Gene-Edited Allograft Lacking CD33, Shows Rapid Primary Engraftment with CD33-Negative Hematopoiesis in Patients with High-Risk Acute Myeloid Leukemia (AML) and Avoids Hematopoietic Toxicity during Gemtuzumab Ozogamicin (GO) Maintenance Post-Hematopoietic Cell Transplant (HCT).

John F. DiPersio, Brenda Cooper, Hyung C. Suh

Regulating PCCA gene expression by modulation of pseudoexon splicing patterns to rescue enzyme activity in propionic acidemia

Ulrika Simone Spangsberg Petersen, Maja Dembić, Ainhoa Martínez‐Pizarro et al.

An AI-driven framework for enhancing regulatory precision and efficiency in CRISPR-Cas gene-edited crops: challenges, opportunities, and global harmonization

Feng Zhu, Zihan Liu, Zeyu Zheng

367 CRISPR/Cas9 gene-edited, allogeneic anti-CD83 CAR-T cells demonstrate potent activity in GvHD and AML tumor models

Ismael Oumzil, Valerie Guerrero, Pooja Smruthi Keerthipati et al.

CRISPR technology towards genome editing of the perennial and semi-perennial crops citrus, coffee and sugarcane

Guilherme Souza Prado, Dhiôvanna Corrêia Rocha, Lucas Nascimento dos Santos et al.

CRISPR technologies for the treatment of Duchenne muscular dystrophy

Eunyoung Choi, Taeyoung Koo

Author Reply to Peer Reviews of CRISPR-Analytics (CRISPR-A): a platform for precise analytics and simulations for gene editing

Marta Sanvicente-García, Albert García-Valiente, Socayna Jouide et al.

<i>CAR1</i>deletion by CRISPR/Cas9 reduces formation of ethyl carbamate from ethanol fermentation by<i>Saccharomyces cerevisiae</i>

Young‐Wook Chin, Woo-Kyung Kang, Hae Won Jang

Cholesterol-rich lipid-mediated nanoparticles boost of transfection efficiency, utilized for gene editing by CRISPR-Cas9

Hosseini ES, M Nikkhah, S Hosseinkhani