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Relevant bibliographies by topics / CRISPR-Cas, CLL, LV

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Journal articles
Dissertations / Theses

Academic literature on the topic 'CRISPR-Cas, CLL, LV'

Author: Grafiati

Published: 14 March 2023

Last updated: 31 July 2025

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Journal articles on the topic "CRISPR-Cas, CLL, LV"

1

Khan, Sikandar. "Recent Advancement and Innovations in CRISPR/Cas and CRISPR Related Technologies: A review." Biotechnology and Bioprocessing 2, no. 5 (2021): 01–12. http://dx.doi.org/10.31579/2766-2314/042.

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CRISPR genome editing technologies have been improving by every passing day. The initial CRISPR/Cas9 technologies, though emerged an improved version of genome editing in competition with TALENS and ZFNs, was nevertheless not free from technical and off-target effects. Technological improvements overtime start addressing issues with original CRISPR/Cas9 technology. The major areas of improvement targeted nucleases and delivery methods. Overtime the nuclease like Cas9 had some modifications like FokI-dCas9, Truncated guide RNAs (tru-gRNAs), Paired Cas9 nickase, Cpf1, Cas6 with Csm/Csr complex a

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2

Dong, Wendy, and Boris Kantor. "Lentiviral Vectors for Delivery of Gene-Editing Systems Based on CRISPR/Cas: Current State and Perspectives." Viruses 13, no. 7 (2021): 1288. http://dx.doi.org/10.3390/v13071288.

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CRISPR/Cas technology has revolutionized the fields of the genome- and epigenome-editing by supplying unparalleled control over genomic sequences and expression. Lentiviral vector (LV) systems are one of the main delivery vehicles for the CRISPR/Cas systems due to (i) its ability to carry bulky and complex transgenes and (ii) sustain robust and long-term expression in a broad range of dividing and non-dividing cells in vitro and in vivo. It is thus reasonable that substantial effort has been allocated towards the development of the improved and optimized LV systems for effective and accurate g

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3

Sun, Wenqiang, Xiaohui Liu, Laichun Song, et al. "The TTN p. Tyr4418Ter mutation causes cardiomyopathy in human and mice." PLOS ONE 19, no. 2 (2024): e0296802. http://dx.doi.org/10.1371/journal.pone.0296802.

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Objective To generate a mouse model carrying TTNtv Y4370* simulating the newly discovered human heterozygous nonsense TTNtv c.13254T>G (p.Tyr4418Ter) to supplement and improve the functional evidence of pathogenic mutation TTNtv c.13254T>G on the pathogenic type of dilated cardiomyopathy. Methods We generated 4 mice carrying TTNtv p. Y4370* through CRISPR/Cas-mediated genome engineering. Monthly serological detection, bimonthly echocardiography, and histology evaluation were carried out to observe and compare alterations of cardiac structure and function between 4 TTN+/- mice and 4 wild-

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4

Suryaprakash, Shruthi, Lei Han, Garret Manquen, et al. "Adenine Base Editing Improves Erythropoiesis in Diamond-Blackfan Anemia Syndrome Patient-Derived Induced Pluripotent Stem Cells." Blood 144, Supplement 1 (2024): 4088. https://doi.org/10.1182/blood-2024-209312.

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Diamond-Blackfan anemia syndrome (DBAS) is an inherited bone marrow failure (BMF) disorder characterized by hypoplastic anemia that presents in infancy. Over time, some patients develop multilineage cytopenias and bone marrow hypocellularity. DBAS is caused by heterozygous loss-of-function mutations in one of 24 ribosomal protein genes, most commonly RPS19. Current therapies such as corticosteroids and red blood cell transfusions are partially effective but have considerable side effects. Hematopoietic stem cell transplantation is curative although many patients lack a suitable donor and/or de

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5

Li, Youngjun, Genshan Ma, Neal Weintraub, and Yaoliang Tang. "Abstract 16620: Enhancing transplanted Cardiac Progenitor Cells homing and survival in Ischemic Heart By CRISPR-dcas9/sgRNA-based Endogenous CXCR4 Promoter Activation." Circulation 132, suppl_3 (2015). http://dx.doi.org/10.1161/circ.132.suppl_3.16620.

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Cardiac progenitor cell (CPC) therapy is promising for repairing the damaged heart, limited stem cell survivability post-transplantation has precluded widespread use of this therapy. Recent reports indicate that interactions between the CXC chemokine stromal cell-derived factor 1 and its receptor CXC chemokine receptor 4 (CXCR4) critically mediate the ischemia-induced recruitment of bone marrow-derived circulating stem/progenitor cells, but the expression of CXCR4 in cardiac progenitor cells is very low. Strategies to overexpress CXCR4 genes in CPC have been shown to improve the survival of do

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Dissertations / Theses on the topic "CRISPR-Cas, CLL, LV"

1

C, Lorenzetti F. "CRISPR/Cpf1 and suicide gene as personalized approach for patients with TP53-mutated Chronic Lymphocytic Leukemia (CLL)." Doctoral thesis, Università di Siena, 2021. http://hdl.handle.net/11365/1133166.

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Chronic Lymphocytic Leukemia (CLL) is a very heterogenous disease caused by alterations in both chromosomes and genes, such as deletion of the 13q14, 11q22-23, 17p12, and trisomy of chromosome 12 or genetic mutations in the TP53, ATM, BRIC3, NOTCH. Within genetic abnormalities, TP53 mutations are detected in a small percentage of leukemia patients (about 10%). Currently adopted therapeutic choices (chemoimmunotherapy, targeted therapy, hematopoietic stem cell transplantation) are not effective due to the acquired abilities of TP53-mutated clones to escape the control systems. For this reason C

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