Combined approaches for increasing fetal hemoglobin (HbF) and de novo production of adult hemoglobin (HbA) in erythroid cells from β-thalassemia patients …

A Finotti, R Gambari - Frontiers in Genome Editing, 2023 - frontiersin.org
Genome editing (GE) is one of the most efficient and useful molecular approaches to correct
the effects of gene mutations in hereditary monogenetic diseases, including β-thalassemia …

[HTML][HTML] CRISPR-based strategies for targeted transgene knock-in and gene correction

CH Lau, C Tin, Y Suh - Faculty Reviews, 2020 - ncbi.nlm.nih.gov
The last few years have seen tremendous advances in CRISPR-mediated genome editing.
Great efforts have been made to improve the efficiency, specificity, editing window, and …

Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients

LC Cosenza, J Gasparello, N Romanini, M Zurlo… - … Therapy Methods & …, 2021 - cell.com
Gene editing by the CRISPR-Cas9 nuclease system technology can be considered among
the most promising strategies to correct hereditary mutations in a variety of monogenic …

A positive, growth-based PAM screen identifies noncanonical motifs recognized by the S. pyogenes Cas9

D Collias, RT Leenay, RA Slotkowski, Z Zuo… - Science …, 2020 - science.org
CRISPR technologies have overwhelmingly relied on the Streptococcus pyogenes Cas9
(SpyCas9), with its consensus NGG and less preferred NAG and NGA protospacer-adjacent …

Co-Treatment of Erythroid Cells from β-Thalassemia Patients with CRISPR-Cas9-Based β039-Globin Gene Editing and Induction of Fetal Hemoglobin

LC Cosenza, C Zuccato, M Zurlo, R Gambari, A Finotti - Genes, 2022 - mdpi.com
Gene editing (GE) is an efficient strategy for correcting genetic mutations in monogenic
hereditary diseases, including β-thalassemia. We have elsewhere reported that CRISPR …

Application of CRISPR-Cas9 editing for virus engineering and the development of recombinant viral vaccines

N Tang, Y Zhang, Z Shen, Y Yao, V Nair - The CRISPR Journal, 2021 - liebertpub.com
CRISPR-Cas technology, discovered originally as a bacterial defense system, has been
extensively repurposed as a powerful tool for genome editing for multiple applications in …

UV radiation enhanced encapsulation of superparamagnetic iron oxide nanoparticles (MNPs) in microparticles derived from tumor repopulating cells

QL Liang, H Liu, T Wang, CH Lau, J Wang… - Biochemical and …, 2024 - Elsevier
Extracellular vesicles (EVs) such as microparticles secreted by the cells can be manipulated
and used for delivering therapeutic drugs to target and eradicate cancer cells. However …

Blood cancer therapy with synthetic receptors and CRISPR technology

H Zhang, M Zhong, J Zhang, C Chen - Leukemia Research, 2025 - Elsevier
Chimeric antigen receptor (CAR)-T and-NK cells showed great success in treating
hematological malignancies, including leukemia, lymphoma, and myeloma. CRISPR …

Recent progress of smart nano-based biosensors and their applications in biomedicine

A Hasanzadeh, HR Azmand, A Ebadati, H Nourizadeh… - Nano, 2022 - World Scientific
Recent advances in miniaturized nano-based devices are rapidly extending the boundaries
of biomedical technologies, particularly biosensors. Highly selective biosensors with the …

With a pinch of salt: metagenomic insights into Namib Desert salt pan microbial mats and halites reveal functionally adapted and competitive communities

L Martínez-Alvarez, JB Ramond, S Vikram… - Applied and …, 2023 - Am Soc Microbiol
Salt pans or playas, which are saline-rich springs surrounded by halite evaporates in arid
environments, have played an essential role in landscape erosion during the formation of …