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 …

Nrf1 promotes heart regeneration and repair by regulating proteostasis and redox balance

M Cui, A Atmanli, MG Morales, W Tan, K Chen… - Nature …, 2021 - nature.com
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 …

[HTML][HTML] A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation

Y Zhang, T Nishiyama, H Li, J Huang, A Atmanli… - … Therapy-Methods & …, 2021 - cell.com
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 …

Cardiac myoediting attenuates cardiac abnormalities in human and mouse models of Duchenne muscular dystrophy

A Atmanli, AC Chai, M Cui, Z Wang… - Circulation …, 2021 - Am Heart Assoc
Rationale: Absence of dystrophin in Duchenne muscular dystrophy (DMD) results in the
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 …

[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 …

Uncovering cellular networks in branching morphogenesis using single-cell transcriptomics

K Goodwin, CM Nelson - Current Topics in Developmental Biology, 2021 - Elsevier
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 …

Live-cell transcriptomics with engineered virus-like particles

MA Najia, J Borrajo, A Le, FN Tsai, JY Huang… - bioRxiv, 2024 - biorxiv.org
The transcriptional state of a cell provides a multi-parameter representation of gene
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 …

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 …