[HTML][HTML] P1429: MULTIPLEX DELETION OF MYELOID ANTIGENS CD33 AND CLL-1 BY CRISPR/CAS9 IN HUMAN HEMATOPOIETIC STEM CELLS HIGHLIGHTS …

J Xavier-Ferrucio, C Luo, G Angelini… - …, 2022 - journals.lww.com
Background: Acute myeloid leukemia (AML) is a heterogeneous disease characterized by
abnormal clonal expansion and is the most common form of adult acute leukemia. Though …

[HTML][HTML] Knock out of CD123 or CLL-1 By CRISPR-Cas9 Editing from Human Hematopoietic Stem Cell Transplants Provide New Possibilities for Increasing …

C Luo, G Angelini, S Krishnamurthy, J Lisle, M Isik… - Blood, 2021 - Elsevier
Acute myeloid leukemia (AML) is a clonal disorder of hematopoiesis and the most common
form of acute leukemia in adults. Most patients with AML relapse despite intensive …

Optimization of CRISPR-Cas9 knock-out of CD33 in human hematopoietic stem/progenitor cells for allogeneic transplantation in patients with acute myeloid leukemia

KD Cummins, J Salas-McKee, I Kulikovskaya, M Gohil… - Cytotherapy, 2019 - Elsevier
Background & Aim Chimeric antigen receptor T-cells (CART) for acute myeloid leukemia
(AML) are limited by hematological toxicity due to shared antigens in AML blasts & healthy …

CRISPR/Cas9 genome editing in human hematopoietic stem cells

RO Bak, DP Dever, MH Porteus - Nature protocols, 2018 - nature.com
Genome editing via homologous recombination (HR)(gene targeting) in human
hematopoietic stem cells (HSCs) has the power to reveal gene–function relationships and …

Understanding genetic heterogeneity in gene-edited hematopoietic stem cell products

HJ Becker, S Yamazaki - Experimental Hematology, 2024 - exphem.org
CRISPR/Cas gene editing has transformed genetic research and is poised to drive the next
generation of gene therapies targeting hematopoietic stem cells (HSCs). However, the …

[PDF][PDF] Efficient Multiplex Gene Editing of CD33 and CLL-1 in Human Hematopoietic Stem Cells Enables the Potential of Next-Generation Transplants for AML …

J Xavier-Ferrucio, G Angelini, S Krishnamurthy, N Patel… - Blood, 2022 - vorbio.com
OBJECTIVES► Demonstrate that multiplex (MPX) deletion of CD33 and CLL-1 from CD34+
human hematopoietic stem and progenitor cells (hHSPCs) does not impact HSC function► …

[HTML][HTML] Understanding Genetic Heterogeneity in Gene-Edited HSC Products

HJ Becker, S Yamazaki - Experimental Hematology, 2023 - Elsevier
CRISPR/Cas gene editing has transformed genetic research and is poised to drive the next
generation of gene therapies targeting hematopoietic stem cells (HSCs). However, the …

[HTML][HTML] Engineering resistance to antigen-specific immunotherapy in normal hematopoietic stem cells by gene editing to enable targeting of acute myeloid leukemia

MY Kim, SS Kenderian, D Schreeder, M Klichinsky… - Blood, 2016 - Elsevier
Acute myeloid leukemia (AML) is challenging to treat with antigen-specific immunotherapy
due to the lack of leukemia-specific surface antigens that are absent from normal …

[HTML][HTML] Non-viral ex vivo genome-editing in mouse bona fide hematopoietic stem cells with CRISPR/Cas9

S Byambaa, H Uosaki, T Ohmori, H Hara… - … Therapy Methods & …, 2021 - cell.com
We conducted two lines of genome-editing experiments of mouse hematopoietic stem cells
(HSCs) with the clustered regularly interspaced short palindromic repeat (CRISPR) and …

Genome editing in clinical practice: A model study for next-gen hematopoietic cell transplants in hematologic malignancies

P Derigs, C Müller-Tidow - Molecular Therapy Methods & Clinical …, 2024 - cell.com
Immunotherapies are precise antigen-specific approaches that revolutionize cancer
treatment. The goal is to specifically target cancer cells while minimizing effects on healthy …