Engineering the delivery system for CRISPR-based genome editing

Z Glass, M Lee, Y Li, Q Xu - Trends in biotechnology, 2018 - cell.com
Clustered regularly interspaced short palindromic repeat-CRISPR-associated protein
(CRISPR-Cas) systems, found in nature as microbial adaptive immune systems, have been …

[HTML][HTML] Computational tools and resources supporting CRISPR-Cas experiments

P Sledzinski, M Nowaczyk, M Olejniczak - Cells, 2020 - mdpi.com
The CRISPR-Cas system has become a cutting-edge technology that revolutionized
genome engineering. The use of Cas9 nuclease is currently the method of choice in most …

[HTML][HTML] De novo mutations in GNAO1, encoding a Gαo subunit of heterotrimeric G proteins, cause epileptic encephalopathy

K Nakamura, H Kodera, T Akita, M Shiina… - The American Journal of …, 2013 - cell.com
Heterotrimeric G proteins, composed of α, β, and γ subunits, can transduce a variety of
signals from seven-transmembrane-type receptors to intracellular effectors. By whole-exome …

[HTML][HTML] Chemical modification of uridine modulates mRNA-mediated proinflammatory and antiviral response in primary human macrophages

H Moradian, T Roch, L Anthofer, A Lendlein… - … Therapy-Nucleic Acids, 2022 - cell.com
In vitro transcribed (IVT)-mRNA has been accepted as a promising therapeutic modality.
Advances in facile and rapid production technologies make IVT-mRNA an appealing …

Electrospun fiber-mediated delivery of neurotrophin-3 mRNA for neural tissue engineering applications

DL Puhl, JL Funnell, TD Fink, A Swaminathan… - Acta Biomaterialia, 2023 - Elsevier
Aligned electrospun fibers provide topographical cues and local therapeutic delivery to
facilitate robust peripheral nerve regeneration. mRNA delivery enables transient expression …

MRNA therapy for Fabry disease

BC Guild, F DeRosa, M Heartlein - US Patent 9,308,281, 2016 - Google Patents
US9308281B2 - MRNA therapy for Fabry disease - Google Patents US9308281B2 - MRNA
therapy for Fabry disease - Google Patents MRNA therapy for Fabry disease Download PDF …

[HTML][HTML] mRNA transfection-induced activation of primary human monocytes and macrophages: dependence on carrier system and nucleotide modification

H Moradian, T Roch, A Lendlein, M Gossen - Scientific reports, 2020 - nature.com
Monocytes and macrophages are key players in maintaining immune homeostasis.
Identifying strategies to manipulate their functions via gene delivery is thus of great interest …

Material design for next-generation mrna vaccines using lipid nanoparticles

A Higuchi, TC Sung, T Wang, QD Ling, SS Kumar… - Polymer …, 2023 - Taylor & Francis
Vaccine development is among the critical issues for ceasing the COVID-19 pandemic. This
review discusses the current usage of biomaterials in vaccine development and provides …

Paving the way towards precise and safe CRISPR genome editing

P Sledzinski, M Dabrowska, M Nowaczyk… - Biotechnology …, 2021 - Elsevier
As the possibilities of CRISPR-Cas9 technology have been revealed, we have entered a
new era of research aimed at increasing its specificity and safety. This stage of technology …

Improved CaP Nanoparticles for Nucleic Acid and Protein Delivery to Neural Primary Cultures and Stem Cells

YW Chao, YL Lee, CS Tseng, LUH Wang, KC Hsia… - ACS …, 2024 - ACS Publications
Efficiently delivering exogenous materials into primary neurons and neural stem cells
(NSCs) has long been a challenge in neurobiology. Existing methods have struggled with …