Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing
F Chemello, AC Chai, H Li, C Rodriguez-Caycedo… - Science …, 2021 - science.org
Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …
dystrophin, which maintains muscle membrane integrity. We used an adenine base editor …
Nrf1 promotes heart regeneration and repair by regulating proteostasis and redox balance
Following injury, cells in regenerative tissues have the ability to regrow. The mechanisms
whereby regenerating cells adapt to injury-induced stress conditions and activate the …
whereby regenerating cells adapt to injury-induced stress conditions and activate the …
[HTML][HTML] A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation
Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene,
is a lethal neuromuscular disease. Correction of DMD mutations in animal models has been …
is a lethal neuromuscular disease. Correction of DMD mutations in animal models has been …
Cardiac myoediting attenuates cardiac abnormalities in human and mouse models of Duchenne muscular dystrophy
Rationale: Absence of dystrophin in Duchenne muscular dystrophy (DMD) results in the
degeneration of skeletal and cardiac muscles. Owing to advances in respiratory …
degeneration of skeletal and cardiac muscles. Owing to advances in respiratory …
Engineering of the Lrp/AsnC‐type transcriptional regulator DecR as a genetically encoded biosensor for multilevel optimization of L‐cysteine biosynthesis pathway in …
Z Zhou, Z Li, Y Zhong, S Xu, Z Li - Biotechnology and …, 2024 - Wiley Online Library
L‐cysteine is an important sulfur‐containing amino acid being difficult to produce by
microbial fermentation. Due to the lack of high‐throughput screening methods, existing …
microbial fermentation. Due to the lack of high‐throughput screening methods, existing …
[HTML][HTML] High cell throughput, programmable fixation reveals the RNA and protein co-regulation with spatially resolved NFκB pseudo-signaling
N Zhang, M Wang, D Nambiar, S Iyer, P Kadakia… - APL …, 2024 - pubs.aip.org
RNA translation to protein is paramount to creating life, yet RNA and protein correlations
vary widely across tissues, cells, and species. To investigate these perplexing results, we …
vary widely across tissues, cells, and species. To investigate these perplexing results, we …
Uncovering cellular networks in branching morphogenesis using single-cell transcriptomics
Single-cell RNA-sequencing (scRNA-seq) and related technologies to identify cell types and
measure gene expression in space, in time, and within lineages have multiplied rapidly in …
measure gene expression in space, in time, and within lineages have multiplied rapidly in …
Live-cell transcriptomics with engineered virus-like particles
The transcriptional state of a cell provides a multi-parameter representation of gene
expression programs that reflect its identity and phenotype. However, current transcriptomic …
expression programs that reflect its identity and phenotype. However, current transcriptomic …
[图书][B] Advances in Bioreactor Design and Multi-Dimensional Analysis for Assessing Maturation Phenotype of Human Engineered Cardiac Tissues
CC van Neste - 2022 - search.proquest.com
Heart failure remains the world's leading cause of death worldwide, with limited treatment
options aside from palliation and a desperate need for effective preclinical models. The …
options aside from palliation and a desperate need for effective preclinical models. The …
Functional genomic and transcriptomic tools for spatial and dynamic phenotypes
HAA Le - 2023 - dspace.mit.edu
Biology is driven by complex cellular processes that require precise regulation in time and in
space. However, the genetic and molecular factors underlying these behaviors are difficult to …
space. However, the genetic and molecular factors underlying these behaviors are difficult to …