Results 51 to 60 of about 25,918 (263)

Adeno‐associated virus serotype 2 capsid variants for improved liver‐directed gene therapy

Hepatology, EarlyView., 2022
Abstract Background and Aims Current liver‐directed gene therapies look for adeno‐associated virus (AAV) vectors with improved efficacy. With this background, capsid engineering is explored. Whereas shuffled capsid library screenings have resulted in potent liver targeting variants with one first vector in human clinical trials, modifying natural ...
Nadja Meumann, Marti Cabanes‐Creus, Moritz Ertelt, Renina Gale Navarro, Julie Lucifora, Qinggong Yuan, Karin Nien‐Huber, Ahmed Abdelrahman, Xuan‐Khang Vu, Liang Zhang, Ann‐Christin Franke, Christian Schmithals, Albrecht Piiper, Annabelle Vogt, Maria Gonzalez‐Carmona, Jochen T. Frueh, Evelyn Ullrich, Philip Meuleman, Steven R. Talbot, Margarete Odenthal, Michael Ott, Erhard Seifried, Clara T. Schoeder, Joachim Schwäble, Leszek Lisowski, Hildegard Büning   +25 more
wiley   +1 more source

Plants regenerated from tissue culture contain stable epigenome changes in rice. [PDF]

, 2013
Most transgenic crops are produced through tissue culture. The impact of utilizing such methods on the plant epigenome is poorly understood. Here we generated whole-genome, single-nucleotide resolution maps of DNA methylation in several regenerated rice ...
Bellizzi, Maria, Ding, Bo, Feng, Suhua, Jacobsen, Steven E, Meyers, Blake C, Pellegrini, Matteo, Simon, Stacey A, Stroud, Hume, Wang, Guo-Liang   +8 more
core   +2 more sources

Cytosine but not adenine base editor generates mutations in mice [PDF]

, 2019
ABSTRACT Deaminase base editing has emerged as a tool to install or correct point mutations in the genomes of living cells in a wide range of organisms and its ultimate success therapeutically depends on its accuracy. Here we have investigated the fidelity of cytosine base editor 4 (BE4) and adenine base editor (ABE) in mouse embryos ...
Lee, Hye Kyung, Smith, Harold E., Liu, Chengyu, Willi, Michaela, Hennighausen, Lothar   +4 more
openaire   +1 more source

Genomics and proteomics: a signal processor's tour [PDF]

, 2004
The theory and methods of signal processing are becoming increasingly important in molecular biology. Digital filtering techniques, transform domain methods, and Markov models have played important roles in gene identification, biological sequence ...
Vaidyanathan, P. P.
core   +2 more sources

Cytosine and adenine deaminase base-editors induce broad and nonspecific changes in gene expression and splicing

Communications Biology, 2021
Jiao Fan, Yige Ding, et al. examine the impact of cytosine and adenine deaminases on transcriptome-wide gene expression and splicing in HeLa and HEK293T cells.
Jiao Fan, Yige Ding, Chao Ren, Ziguo Song, Jie Yuan, Qiuzhen Chen, Chenchen Du, Chao Li, Xiaolong Wang, Wenjie Shu   +9 more
doaj   +1 more source

Methylation status of Vitamin D receptor gene promoter in benign and malignant adrenal tumors [PDF]

, 2015
We previously showed a decreased expression of vitamin D receptor (VDR) mRNA/protein in a small group of adrenocortical carcinoma (ACC) tissues, suggesting the loss of a protective role of VDR against malignant cell growth in this cancer type ...
Cappellesso, Rocco, Fallo, Francesco, Fassina, Ambrogio, Guzzardo, Vincenza, Pilon, Catia, Rebellato, Andrea, Sasano, Hironobu, Urbanet, Riccardo   +7 more
core   +10 more sources

Two Compact Cas9 Ortholog-Based Cytosine Base Editors Expand the DNA Targeting Scope and Applications In Vitro and In Vivo

Frontiers in Cell and Developmental Biology, 2022
CRISPR/Cas9-based base editing tools enable precise genomic installation and hold great promise for gene therapy, whereas the big size of Cas9 nucleases and its reliability on specific protospacer adjacent motif (PAM) sequences as well as target site ...
Susu Wu, Liping Li, Min Li, Shiyu Sun, Shiyu Sun, Yuting Zhao, Xiaowen Xue, Feiyu Chen, Jingli Zhong, Junfan Guo, Junfan Guo, Qianhui Qu, Xiongjun Wang, Zhen Liu, Zhen Liu, Yunbo Qiao   +15 more
doaj   +1 more source

Maternal nutritional status, C1 metabolism and offspring DNA methylation: a review of current evidence in human subjects. [PDF]

, 2011
: Evidence is growing for the long-term effects of environmental factors during early-life on later disease susceptibility. It is believed that epigenetic mechanisms (changes in gene function not mediated by DNA sequence alteration), particularly DNA ...
Cox, SE, Dominguez-Salas, P, Hennig, BJ, Moore, SE, Prentice, AM   +4 more
core   +4 more sources

Genome Engineering in Plant Using an Efficient CRISPR-xCas9 Toolset With an Expanded PAM Compatibility

Frontiers in Genome Editing, 2020
The CRISPR-Cas9 system enables simple, rapid, and effective genome editing in many species. Nevertheless, the requirement of an NGG protospacer adjacent motif (PAM) for the widely used canonical Streptococcus pyogenes Cas9 (SpCas9) limits the potential ...
Chengwei Zhang, Guiting Kang, Xinxiang Liu, Si Zhao, Shuang Yuan, Lu Li, Yongxing Yang, Feipeng Wang, Xiang Zhang, Jinxiao Yang   +9 more
doaj   +1 more source

Precise genome editing with base editors

Medical Review, 2023
Single-nucleotide variants account for about half of known pathogenic genetic variants in human. Genome editing strategies by reversing pathogenic point mutations with minimum side effects have great therapeutic potential and are now being actively ...
Liu Hongcai, Zhu Yao, Li Minjie, Gu Zhimin   +3 more
doaj   +1 more source