Results 251 to 260 of about 361,542 (380)

PERCC1‐associated enteropathy: Diagnostic challenges and enteral autonomy achieved with teduglutide

JPGN Reports, EarlyView.
Abstract Congenital diarrheas and enteropathies (CODE) are rare inherited disorders characterized by early‐onset intractable diarrhea. Though progress has been made in elucidating the genetic basis of CODE, much remains to be discovered. Another challenge is the lack of curative therapies—treatment is primarily supportive including enteral and ...
Angela Tran, Vivien Nguyen, Phuong Huynh
wiley   +1 more source

Enhancement of therapeutic transgene insertion for treatment of murine phenylketonuria. [PDF]

Mol Ther Nucleic Acids
Martinez MA, Richards DY, Winn SR, Baris AM, Vonada A, Harper L, Harding CO.   +6 more
europepmc   +1 more source

The regulatory frameworks surrounding CRISPR‐edited papaya and their impact on international commerce

Journal of the Science of Food and Agriculture, EarlyView.
Abstract The papaya tree (Carica papaya L.), native to the Americas, is cultivated in tropical regions and holds substantial economic importance, with an estimated export volume of 365 000 t in 2023. However, diseases caused by viruses, fungi, bacteria, and nematodes can lead to severe losses.
Luíza Favaratto, Mirielson L da Silva, David S Buss, Oeber F Quadros, Raul Tapia‐Tussell, José A Ventura, Antonio Alberto R Fernandes, Patricia M B Fernandes   +7 more
wiley   +1 more source

Protocol for mapping insertion sites of Tol2 transgenes in zebrafish using TransTag. [PDF]

STAR Protoc
Wei X, Naidu V, Schneider P, Murphy PJ, Meng FW.   +4 more
europepmc   +1 more source

The gypsy Insulator of Drosophila melanogaster, Together With Its Binding Protein Suppressor of Hairy-Wing, Facilitate High and Precise Expression of Transgenes in Arabidopsis thaliana [PDF]

, 2010
Wenjing She, Weiqiang Lin, Yubin Zhu, Yong Chen, Weiyuan Jin, Yanjun Yang, Ning Han, Hongwu Bian, Muyuan Zhu, Junhui Wang   +9 more
openalex   +1 more source

A conditional tissue-specific transgene expression system using inducible GAL4

Proceedings of the National Academy of Sciences of the United States of America, 2001
Thomas Osterwalder, Kenneth S. Yoon, B. White, H. Keshishian   +3 more
semanticscholar   +1 more source

Autophagy Plays a Suppressive Role in Bladder Tumor Formation in an Orthotopic Mouse Model and Bladder Cancer Patient Specimens

The Kaohsiung Journal of Medical Sciences, EarlyView.
ABSTRACT Autophagy plays either a suppressing or promoting role during tumor development. Clarifying the role of autophagy in bladder tumorigenesis both in vitro and in vivo is crucial for developing novel therapeutic strategies through manipulating autophagy activity.
Wan‐Ting Kuo, Chin‐Chen Pan, Yi‐Wen Liu, Nan‐Haw Chow, Hong‐Lin Cheng, Shan‐Ying Wu, Sheng‐Hui Lan, Chih‐Peng Chang, Hsiao‐Sheng Liu   +8 more
wiley   +1 more source

Balancing Promise and Peril: Hemophilia Gene Therapy Insights. [PDF]

IUBMB Life
Akula S, Borroni E, Cottonaro A, Follenzi A, Merlin S.   +4 more
europepmc   +1 more source

CRISPR Enabled Precision Oncology: From Gene Editing to Tumor Microenvironment Remodeling

Med Research, EarlyView.
CRISPR technology has progressed from a prokaryotic immune system to a diverse suite of editing platforms, including Cas nucleases, base and prime editors, and RNA‐targeting enzymes. These advances enable precise genomic and epigenomic interventions, high‐throughput functional screening, and immune engineering.
Kailai Li, Peixin Huang, Yue Qian, Anqi Lin, Jingjun He, Junyi Shen, Li Chen, Kai Miao, Jian Zhang   +8 more
wiley   +1 more source

Gene Therapy in Crohn's Disease: Current Preclinical Challenges and Future Translational Avenues. [PDF]

Biomedicines
Abdalla S, Brouquet A, Aron-Badin R, Bougnères P.   +3 more
europepmc   +1 more source