Prime editing: advances and therapeutic applications
Z Zhao, P Shang, P Mohanraju, N Geijsen - Trends in Biotechnology, 2023 - cell.com
Clustered regularly interspaced short palindromic repeats-associated protein 9 (CRISPR–
Cas)-mediated genome editing has revolutionized biomedical research and will likely …
Cas)-mediated genome editing has revolutionized biomedical research and will likely …
Therapy development by genome editing of hematopoietic stem cells
L Koniali, CW Lederer, M Kleanthous - Cells, 2021 - mdpi.com
Accessibility of hematopoietic stem cells (HSCs) for the manipulation and repopulation of the
blood and immune systems has placed them at the forefront of cell and gene therapy …
blood and immune systems has placed them at the forefront of cell and gene therapy …
Editing human hematopoietic stem cells: advances and challenges
Genome editing of hematopoietic stem and progenitor cells is being developed for the
treatment of several inherited disorders of the hematopoietic system. The adaptation of …
treatment of several inherited disorders of the hematopoietic system. The adaptation of …
Challenges for gene editing in common variable immunodeficiency disorders: Current and future prospects
R Ameratunga, E Leung, ST Woon, E Lea, C Allan… - Clinical …, 2023 - Elsevier
The original CRISPR Cas9 gene editing system and subsequent innovations offers
unprecedented opportunities to correct severe genetic defects including those causing …
unprecedented opportunities to correct severe genetic defects including those causing …
Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47phox-deficient chronic granulomatous disease
p47 phox-deficient chronic granulomatous disease (p47-CGD) is a primary
immunodeficiency caused by mutations in the neutrophil cytosolic factor 1 (NCF1) gene …
immunodeficiency caused by mutations in the neutrophil cytosolic factor 1 (NCF1) gene …
CRISPR-Cas9 based genome editing for defective gene correction in humans and other mammals
Clustered regularly interspaced short palindromic repeat-Cas9 (CRISPR/Cas9), derived
from bacterial and archean immune systems, has received much attention from the scientific …
from bacterial and archean immune systems, has received much attention from the scientific …
[HTML][HTML] Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47phox-deficient chronic granulomatous disease
KM Siow, M Güngör, D Wrona, F Raimondi… - Molecular Therapy …, 2024 - ncbi.nlm.nih.gov
Abstract p47 phox-deficient chronic granulomatous disease (p47-CGD) is a primary
immunodeficiency caused by mutations in the neutrophil cytosolic factor 1 (NCF1) gene …
immunodeficiency caused by mutations in the neutrophil cytosolic factor 1 (NCF1) gene …
Unexpectedly high levels of inverted re-insertions using paired sgRNAs for genomic deletions
J Blayney, EM Foster, M Jagielowicz, M Kreuzer… - Methods and …, 2020 - mdpi.com
Use of dual sgRNAs is a common CRISPR/Cas9-based strategy for the creation of genetic
deletions. The ease of screening combined with a rather high rate of success makes this …
deletions. The ease of screening combined with a rather high rate of success makes this …
[HTML][HTML] 基因编辑治疗原发性免疫缺陷病
刘珊 - Chinese Journal of Contemporary Pediatrics, 2021 - ncbi.nlm.nih.gov
基因编辑是基于人工核酸酶的先进技术， 可对基因组序列进行精确修饰。 基因编辑在医药领域
展现了巨大的应用前景， 为疾病的治疗提供了新的精准治疗方法。 原发性免疫缺陷病是单基因 …
展现了巨大的应用前景， 为疾病的治疗提供了新的精准治疗方法。 原发性免疫缺陷病是单基因 …
Gene editing for the treatment of primary immunodeficiency disease
S Liu, SY Fang, YF An - Zhongguo Dang dai er ke za zhi= Chinese …, 2021 - europepmc.org
基因编辑是基于人工核酸酶的先进技术, 可对基因组序列进行精确修饰. 基因编辑在医药领域
展现了巨大的应用前景, 为疾病的治疗提供了新的精准治疗方法. 原发性免疫缺陷病是单基因 …
展现了巨大的应用前景, 为疾病的治疗提供了新的精准治疗方法. 原发性免疫缺陷病是单基因 …