Engineering precision nanoparticles for drug delivery

MJ Mitchell, MM Billingsley, RM Haley… - Nature reviews drug …, 2021 - nature.com
In recent years, the development of nanoparticles has expanded into a broad range of
clinical applications. Nanoparticles have been developed to overcome the limitations of free …

Exploring the trans‐cleavage activity of CRISPR‐Cas12a (cpf1) for the development of a universal electrochemical biosensor

Y Dai, RA Somoza, L Wang, JF Welter, Y Li… - Angewandte …, 2019 - Wiley Online Library
An accurate, rapid, and cost‐effective biosensor for the quantification of disease biomarkers
is vital for the development of early‐diagnostic point‐of‐care systems. The recent discovery …

Cystic fibrosis in the year 2020: A disease with a new face

K De Boeck - Acta paediatrica, 2020 - Wiley Online Library
The autosomal recessive disease cystic fibrosis (CF) was once untreatable and deadly in
childhood, but now most patients survive to adulthood. Many countries have instituted CF …

Secretory cells dominate airway CFTR expression and function in human airway superficial epithelia

K Okuda, H Dang, Y Kobayashi, G Carraro… - American journal of …, 2021 - atsjournals.org
Rationale: Identification of the specific cell types expressing CFTR (cystic fibrosis [CF]
transmembrane conductance regulator) is required for precision medicine therapies for CF …

RNA‐based medicine: from molecular mechanisms to therapy

A Sparmann, J Vogel - The EMBO Journal, 2023 - embopress.org
RNA‐based therapeutics have the potential to revolutionize the treatment and prevention of
human diseases. While early research faced setbacks, it established the basis for …

CRISPR-Cas tools and their application in genetic engineering of human stem cells and organoids

D Hendriks, H Clevers, B Artegiani - Cell stem cell, 2020 - cell.com
CRISPR-Cas technology has revolutionized biological research and holds great therapeutic
potential. Here, we review CRISPR-Cas systems and their latest developments with an …

Gene therapy for cystic fibrosis: new tools for precision medicine

JA Lee, A Cho, EN Huang, Y Xu, H Quach, J Hu… - Journal of Translational …, 2021 - Springer
The discovery of the Cystic fibrosis (CF) gene in 1989 has paved the way for incredible
progress in treating the disease such that the mean survival age of individuals living with CF …

Mechanism-based traps enable protease and hydrolase substrate discovery

S Tang, AT Beattie, L Kafkova, G Petris… - Nature, 2022 - nature.com
Hydrolase enzymes, including proteases, are encoded by 2–3% of the genes in the human
genome and 14% of these enzymes are active drug targets. However, the activities and …

Application of CRISPR/Cas9 to human-induced pluripotent stem cells: from gene editing to drug discovery

C De Masi, P Spitalieri, M Murdocca, G Novelli… - Human genomics, 2020 - Springer
Human-induced pluripotent stem cells (hiPSCs) and CRISPR/Cas9 gene editing system
represent two instruments of basic and translational research, which both allow to acquire …

Long-term differentiating primary human airway epithelial cell cultures: how far are we?

Z Bukowy-Bieryłło - Cell Communication and Signaling, 2021 - Springer
Background Human airway epithelial (HAE) cellular models are widely used in applicative
studies of the airway physiology and disease. In vitro expanded and differentiated primary …