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Gene therapy for inborn errors of immunity: past, present and future
A Fischer - Nature Reviews Immunology, 2023 - nature.com
Inborn errors of immunity (IEI) are diseases caused by genetic mutations that affect the
immune system's ability to fight pathogens, cope with the microbiota or regulate …
immune system's ability to fight pathogens, cope with the microbiota or regulate …
CRISPR-based gene therapies: from preclinical to clinical treatments
In recent years, clustered regularly interspaced short palindromic repeats (CRISPRs) and
CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat …
CRISPR-associated (Cas) protein have emerged as a revolutionary gene editing tool to treat …
Peptide-mediated delivery of CRISPR enzymes for the efficient editing of primary human lymphocytes
DV Foss, JJ Muldoon, DN Nguyen, D Carr… - Nature biomedical …, 2023 - nature.com
CRISPR-mediated genome editing of primary human lymphocytes is typically carried out via
electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the …
electroporation, which can be cytotoxic, cumbersome and costly. Here we show that the …
Epitope editing enables targeted immunotherapy of acute myeloid leukaemia
G Casirati, A Cosentino, A Mucci, M Salah Mahmoud… - Nature, 2023 - nature.com
Despite the considerable efficacy observed when targeting a dispensable lineage antigen,
such as CD19 in B cell acute lymphoblastic leukaemia,, the broader applicability of adoptive …
such as CD19 in B cell acute lymphoblastic leukaemia,, the broader applicability of adoptive …
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
Lipid nanoparticles allow efficient and harmless ex vivo gene editing of human hematopoietic cells
Ex vivo gene editing in T cells and hematopoietic stem/progenitor cells (HSPCs) holds
promise for treating diseases. Gene editing encompasses the delivery of a programmable …
promise for treating diseases. Gene editing encompasses the delivery of a programmable …
[HTML][HTML] Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells
A Omer-Javed, G Pedrazzani, L Albano, S Ghaus… - Cell, 2022 - cell.com
Hematopoietic stem/progenitor cell gene therapy (HSPC-GT) is proving successful to treat
several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo …
several genetic diseases. HSPCs are mobilized, harvested, genetically corrected ex vivo …
[HTML][HTML] Advances in gene therapy for inborn errors of immunity
LMO de Bruin, AC Lankester… - Current Opinion in Allergy …, 2023 - journals.lww.com
Gene therapy by modifying autologous hematopoietic stem cells (HSCs) offers an attractive
alternative to allogeneic hematopoietic stem cell transplantation (HSCT), the current …
alternative to allogeneic hematopoietic stem cell transplantation (HSCT), the current …
CRISPR/Cas9 ribonucleoprotein-mediated genome and epigenome editing in mammalian cells
H Bloomer, J Khirallah, Y Li, Q Xu - Advanced drug delivery reviews, 2022 - Elsevier
The clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated
protein (Cas) system has revolutionized the ability to edit the mammalian genome, providing …
protein (Cas) system has revolutionized the ability to edit the mammalian genome, providing …
Therapeutic gene editing of T cells to correct CTLA-4 insufficiency
TA Fox, BC Houghton, L Petersone, E Waters… - Science Translational …, 2022 - science.org
Heterozygous mutations in CTLA-4 result in an inborn error of immunity with an autoimmune
and frequently severe clinical phenotype. Autologous T cell gene therapy may offer a cure …
and frequently severe clinical phenotype. Autologous T cell gene therapy may offer a cure …