Systematic optimization of prime editing for the efficient functional correction of CFTR F508del in human airway epithelial cells
Prime editing (PE) enables precise and versatile genome editing without requiring double-
stranded DNA breaks. Here we describe the systematic optimization of PE systems to …
stranded DNA breaks. Here we describe the systematic optimization of PE systems to …
Delivering the CRISPR/Cas9 system for engineering gene therapies: Recent cargo and delivery approaches for clinical translation
RA Foley, RA Sims, EC Duggan, JK Olmedo… - … in Bioengineering and …, 2022 - frontiersin.org
Clustered Regularly Interspaced Short Palindromic Repeats associated protein 9
(CRISPR/Cas9) has transformed our ability to edit the human genome selectively. This …
(CRISPR/Cas9) has transformed our ability to edit the human genome selectively. This …
[HTML][HTML] Genomic Modifications of Lactic Acid Bacteria and Their Applications in Dairy Fermentation
Z Xie, O McAuliffe, YS Jin, MJ Miller - Journal of Dairy Science, 2024 - Elsevier
ABSTRACT Lactic Acid Bacteria (LAB) have a long history of safe use in milk fermentation
and are generally recognized as health-promoting microorganisms when present in …
and are generally recognized as health-promoting microorganisms when present in …
In vivo editing of lung stem cells for durable gene correction in mice
In vivo genome correction holds promise for generating durable disease cures; yet, effective
stem cell editing remains challenging. In this work, we demonstrate that optimized lung …
stem cell editing remains challenging. In this work, we demonstrate that optimized lung …
Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo
H Zhang, K Kelly, J Lee, D Echeverria… - Nucleic acids …, 2024 - academic.oup.com
Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add
challenges for delivery. Chemical modification, which has been key to the success of …
challenges for delivery. Chemical modification, which has been key to the success of …
Chemical engineering of CRISPR–Cas systems for therapeutic application
Clustered regularly interspaced short palindromic repeats (CRISPR) technology has
transformed molecular biology and the future of gene-targeted therapeutics. CRISPR …
transformed molecular biology and the future of gene-targeted therapeutics. CRISPR …
Gene editing in common cardiovascular diseases
AM Lauerer, XM Caravia, LS Maier, F Chemello… - Pharmacology & …, 2024 - Elsevier
Cardiovascular diseases are the leading cause of morbidity and mortality worldwide,
highlighting the high socioeconomic impact. Current treatment strategies like compound …
highlighting the high socioeconomic impact. Current treatment strategies like compound …
Vector enabled CRISPR gene editing–A revolutionary strategy for targeting the diversity of brain pathologies
Brain pathologies are considered one of the greatest contributors of death and disability
worldwide. Neurodegenerative Alzheimer's disease is the second leading cause of death in …
worldwide. Neurodegenerative Alzheimer's disease is the second leading cause of death in …
Influence of N1-Methylpseudouridine in Guide RNAs on CRISPR/Cas9 Activity
D Prokhorova, A Matveeva, A Zakabunin… - International Journal of …, 2023 - mdpi.com
At present, there are many strategies to improve the activity of CRISPR/Cas9. A well-known
and effective approach is guide RNA modification. Many chemical guide RNA modifications …
and effective approach is guide RNA modification. Many chemical guide RNA modifications …
Genome editing using type IE CRISPR-Cas3 in mice and rat zygotes
The type I CRISPR system has recently emerged as a promising tool, especially for large-
scale genomic modification, but its application to generate model animals by editing zygotes …
scale genomic modification, but its application to generate model animals by editing zygotes …