Manipulation of developmental gamma-globin gene expression: an approach for healing hemoglobinopathies
ABSTRACT β-Hemoglobinopathies are the most common monogenic disorders, and a
century of research has provided us with a better understanding of the attributes of these …
century of research has provided us with a better understanding of the attributes of these …
Current and future gene therapies for hemoglobinopathies
C Brendel, DA Williams - Current opinion in hematology, 2020 - journals.lww.com
Early short-term therapeutic benefit has been reported for some of the ongoing clinical trials,
but confirmation of long-term safety and efficacy remains to be shown. Future therapies …
but confirmation of long-term safety and efficacy remains to be shown. Future therapies …
Predicting base editing outcomes using position-specific sequence determinants
CRISPR/Cas base editors promise nucleotide-level control over DNA sequences, but the
determinants of their activity remain incompletely understood. We measured base editing …
determinants of their activity remain incompletely understood. We measured base editing …
Clinical and haematological characteristics of 38 individuals with Hb G‐Makassar in Malaysia
Abstract Haemoglobin (Hb) G‐Makassar is a rare Hb variant. It presents a diagnostic
challenge as it imitates sickle Hb (Hb S) in standard electrophoresis and high‐performance …
challenge as it imitates sickle Hb (Hb S) in standard electrophoresis and high‐performance …
Alpha-1 antitrypsin deficiency and recombinant protein sources with focus on plant sources: Updates, challenges and perspectives
MJ McNulty, DZ Silberstein, BT Kuhn… - Free Radical Biology …, 2021 - Elsevier
Abstract Alpha-1 antitrypsin deficiency (A1ATD) is an autosomal recessive disease
characterized by low plasma levels of A1AT, a serine protease inhibitor representing the …
characterized by low plasma levels of A1AT, a serine protease inhibitor representing the …
Quantitative modelling of CRISPR-Cas editing outcomes
A Pallaseni - 2023 - repository.cam.ac.uk
Abstract Development of the CRISPR-Cas toolkit over the last decade has enabled
unprecedented control over the genome and unlocked the capacity for new experiments and …
unprecedented control over the genome and unlocked the capacity for new experiments and …
DNA Base Editing Strategies for Genome Editing
M Coelho, S Li, BJM Taylor - Genome Editing in Drug …, 2022 - Wiley Online Library
The choice of base editing technology is critical for the success of a gene editing experiment
but can seem overwhelming, as each component of the base editing machinery has been …
but can seem overwhelming, as each component of the base editing machinery has been …
Correction of the Sickle Cell Mutation Through Base and Prime Editing in Hematopoietic Stem Cells
K Ji - 2020 - preprints.org
Sickle cell disease is characterized by stiff,“sickled” red blood cells that have difficulty
moving through the bloodstream and do not efficiently carry oxygen. It is an inherited …
moving through the bloodstream and do not efficiently carry oxygen. It is an inherited …
[PDF][PDF] Precise genomic deletions and insertions via paired prime editing for crop bioengineering
JL Moreno-Ramírez - 2022 - repository.kaust.edu.sa
CRISPR/Cas has been developed for targeted mutagenesis in diverse species, including
plants. However, precise genome editing via homology-directed repair (HDR) is inefficient in …
plants. However, precise genome editing via homology-directed repair (HDR) is inefficient in …
[PDF][PDF] Precise genomic deletions and insertions via paired prime editing for crop bioengineering
JLM Ramirez - 2022 - repository.kaust.edu.sa
ABSTRACT CRISPR/Cas has been developed for targeted mutagenesis in diverse species,
including plants. However, precise genome editing via homology-directed repair (HDR) is …
including plants. However, precise genome editing via homology-directed repair (HDR) is …