Dual intron-targeted CRISPR-Cas9-mediated disruption of the AML RUNX1-RUNX1T1 fusion gene effectively inhibits proliferation and decreases tumor volume in vitro and in vivo

CRISPRon/off: CRISPR/Cas9 on- and off-target gRNA design

(2022) Christian Anthon et al.   BIOINFORMATICS

Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice

(2021) Adam C. Wilkinson et al.   Nature Communications

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

(2021) Toni S. Pearson et al.   Nature Communications

CD33 Expression and Gentuzumab Ozogamicin in Acute Myeloid Leukemia: Two Sides of the Same Coin

(2021) Matteo Molica et al.   Cancers

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

(2021) Eriya Kenjo et al.   Nature Communications

Involvement of classic and alternative non-homologous end joining pathways in hematologic malignancies: targeting strategies for treatment

(2021) Mohsen Valikhani et al.   Experimental Hematology & Oncology

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

CAR T Cells for Acute Myeloid Leukemia: State of the Art and Future Directions

(2020) Sherly Mardiana et al.   Frontiers in Oncology

In vivo CRISPR/Cas9 targeting of fusion oncogenes for selective elimination of cancer cells

(2020) M. Martinez-Lage et al.   Nature Communications

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia

(2020) Haydar Frangoul et al.   NEW ENGLAND JOURNAL OF MEDICINE

Precise Gene Editing Preserves Hematopoietic Stem Cell Function following Transient p53-Mediated DNA Damage Response

(2019) Giulia Schiroli et al.   Cell Stem Cell

Epidemiology of acute myeloid leukemia: Recent progress and enduring challenges

(2019) Rory M. Shallis et al.   BLOOD REVIEWS

RUNX1-ETO Depletion in t(8;21) AML Leads to C/EBPα- and AP-1-Mediated Alterations in Enhancer-Promoter Interaction

(2019) Anetta Ptasinska et al.   Cell Reports

Engineering the AAV capsid to evade immune responses

(2019) Christopher Barnes et al.   CURRENT OPINION IN BIOTECHNOLOGY

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

(2018) Christopher A. Vakulskas et al.   NATURE MEDICINE

Imprecision and DNA Break Repair Biased towards Incompatible End Joining in Leukemia

(2017) Franz Josef Gassner et al.   MOLECULAR CANCER RESEARCH

Trends in hematological cancer in the elderly in Denmark, 1980–2012

(2016) Lukas F. Ocias et al.   ACTA ONCOLOGICA

Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel

(2016) Hartmut Döhner et al.   BLOOD

The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia

(2016) D. A. Arber et al.   BLOOD

CrispRVariants charts the mutation spectrum of genome engineering experiments

(2016) Helen Lindsay et al.   NATURE BIOTECHNOLOGY

The genomic landscape of core-binding factor acute myeloid leukemias

(2016) Zachary J Faber et al.   NATURE GENETICS

Core-binding factor acute myeloid leukemia in first relapse: a retrospective study from the French AML Intergroup

(2014) M.-A. Hospital et al.   BLOOD

Addition of gemtuzumab ozogamicin to induction chemotherapy in adult patients with acute myeloid leukaemia: a meta-analysis of individual patient data from randomised controlled trials

(2014) Robert K Hills et al.   LANCET ONCOLOGY

Prospective evaluation of gene mutations and minimal residual disease in patients with core binding factor acute myeloid leukemia

(2013) E. Jourdan et al.   BLOOD

Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

(2013) Seung Woo Cho et al.   NATURE BIOTECHNOLOGY

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Addiction of t(8;21) and inv(16) Acute Myeloid Leukemia to Native RUNX1

(2013) Oren Ben-Ami et al.   Cell Reports

Minimal residual disease monitoring by quantitative RT-PCR in core binding factor AML allows risk stratification and predicts relapse: results of the United Kingdom MRC AML-15 trial

(2012) J. A. L. Yin et al.   BLOOD

Refinement of cytogenetic classification in acute myeloid leukemia: determination of prognostic significance of rare recurring chromosomal abnormalities among 5876 younger adult patients treated in the United Kingdom Medical Research Council trials

(2010) D. Grimwade et al.   BLOOD

Acute Myeloid Leukemia With Translocation (8;21) or Inversion (16) in Elderly Patients Treated With Conventional Chemotherapy: A Collaborative Study of the French CBF-AML Intergroup

(2009) Thomas Prébet et al.   JOURNAL OF CLINICAL ONCOLOGY