Literature DB >> 31003997

CRISPR/Cas9 PIG -A gene editing in nonhuman primate model demonstrates no intrinsic clonal expansion of PNH HSPCs.

Tae-Hoon Shin1, Eun Jung Baek1,2, Marcus A F Corat1,3, Shirley Chen1, Jean-Yves Metais1,4, Aisha A AlJanahi1, Yifan Zhou1, Robert E Donahue1, Kyung-Rok Yu1,5, Cynthia E Dunbar1.   

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Year:  2019        PMID： 31003997      PMCID： PMC6557622          DOI： 10.1182/blood.2019000800

Source DB:  PubMed          Journal:  Blood        ISSN： 0006-4971            Impact factor:   22.113


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  23 in total

1.  Genetic and environmental effects in paroxysmal nocturnal hemoglobinuria: this little PIG-A goes "Why? Why? Why?".

Authors:  N S Young; J P Maciejewski
Journal:  J Clin Invest       Date:  2000-09       Impact factor: 14.808

2.  Murine embryonic stem cells without pig-a gene activity are competent for hematopoiesis with the PNH phenotype but not for clonal expansion.

Authors:  V Rosti; G Tremml; V Soares; P P Pandolfi; L Luzzatto; M Bessler
Journal:  J Clin Invest       Date:  1997-09-01       Impact factor: 14.808

3.  Quantitative stability of hematopoietic stem and progenitor cell clonal output in rhesus macaques receiving transplants.

Authors:  Samson J Koelle; Diego A Espinoza; Chuanfeng Wu; Jason Xu; Rong Lu; Brian Li; Robert E Donahue; Cynthia E Dunbar
Journal:  Blood       Date:  2017-01-13       Impact factor: 22.113

4.  Genetic Inactivation of CD33 in Hematopoietic Stem Cells to Enable CAR T Cell Immunotherapy for Acute Myeloid Leukemia.

Authors:  Miriam Y Kim; Kyung-Rok Yu; Saad S Kenderian; Marco Ruella; Shirley Chen; Tae-Hoon Shin; Aisha A Aljanahi; Daniel Schreeder; Michael Klichinsky; Olga Shestova; Miroslaw S Kozlowski; Katherine D Cummins; Xinhe Shan; Maksim Shestov; Adam Bagg; Jennifer J D Morrissette; Palak Sekhri; Cicera R Lazzarotto; Katherine R Calvo; Douglas B Kuhns; Robert E Donahue; Gregory K Behbehani; Shengdar Q Tsai; Cynthia E Dunbar; Saar Gill
Journal:  Cell       Date:  2018-05-31       Impact factor: 41.582

Review 5.  Somatic mutations in paroxysmal nocturnal hemoglobinuria: a blessing in disguise?

Authors:  L Luzzatto; M Bessler; B Rotoli
Journal:  Cell       Date:  1997-01-10       Impact factor: 41.582

6.  Resistance to apoptosis caused by PIG-A gene mutations in paroxysmal nocturnal hemoglobinuria.

Authors:  R A Brodsky; M S Vala; J P Barber; M E Medof; R J Jones
Journal:  Proc Natl Acad Sci U S A       Date:  1997-08-05       Impact factor: 11.205

7.  PIG-A mutations in normal hematopoiesis.

Authors:  Rong Hu; Galina L Mukhina; Steven Piantadosi; Jamie P Barber; Richard J Jones; Robert A Brodsky
Journal:  Blood       Date:  2005-02-01       Impact factor: 22.113

8.  Genomic safe harbors permit high β-globin transgene expression in thalassemia induced pluripotent stem cells.

Authors:  Eirini P Papapetrou; Gabsang Lee; Nirav Malani; Manu Setty; Isabelle Riviere; Laxmi M S Tirunagari; Kyuichi Kadota; Shoshannah L Roth; Patricia Giardina; Agnes Viale; Christina Leslie; Frederic D Bushman; Lorenz Studer; Michel Sadelain
Journal:  Nat Biotechnol       Date:  2010-12-12       Impact factor: 54.908

9.  Repair of double-strand breaks induced by CRISPR-Cas9 leads to large deletions and complex rearrangements.

Authors:  Michael Kosicki; Kärt Tomberg; Allan Bradley
Journal:  Nat Biotechnol       Date:  2018-07-16       Impact factor: 54.908

10.  Dynamics of hematopoiesis in paroxysmal nocturnal hemoglobinuria (PNH): no evidence for intrinsic growth advantage of PNH clones.

Authors:  D J Araten; M Bessler; S McKenzie; H Castro-Malaspina; B H Childs; F Boulad; A Karadimitris; R Notaro; L Luzzatto
Journal:  Leukemia       Date:  2002-11       Impact factor: 11.528
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  8 in total

1.  Secondary myelodysplastic syndrome and leukemia in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria.

Authors:  Lova Sun; Daria V Babushok
Journal:  Blood       Date:  2020-07-02       Impact factor: 22.113

Review 2.  When does a PNH clone have clinical significance?

Authors:  Daria V Babushok
Journal:  Hematology Am Soc Hematol Educ Program       Date:  2021-12-10

Review 3.  Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges.

Authors:  Jenna K Schmidt; Kathryn M Jones; Trevor Van Vleck; Marina E Emborg
Journal:  Sci Transl Med       Date:  2022-03-02       Impact factor: 19.319

4.  Paroxysmal nocturnal hemoglobinuria caused by CN-LOH of constitutional PIGB mutation and 70-kbp microdeletion on 15q.

Authors:  Saskia Langemeijer; Charlotte Schaap; Frank Preijers; Joop H Jansen; Nicole Blijlevens; Norimitsu Inoue; Petra Muus; Taroh Kinoshita; Yoshiko Murakami
Journal:  Blood Adv       Date:  2020-11-24

Review 5.  Understanding and overcoming adverse consequences of genome editing on hematopoietic stem and progenitor cells.

Authors:  Byung-Chul Lee; Richard J Lozano; Cynthia E Dunbar
Journal:  Mol Ther       Date:  2021-09-10       Impact factor: 11.454

6.  A Pig-a conditional knock-out mice model mediated by Vav-iCre: stable GPI-deficient and mild hemolysis.

Authors:  Yingying Chen; Hui Liu; Lijie Zeng; Liyan Li; Dan Lu; Zhaoyun Liu; Rong Fu
Journal:  Exp Hematol Oncol       Date:  2022-01-15

Review 7.  Insights Into the Emergence of Paroxysmal Nocturnal Hemoglobinuria.

Authors:  Melissa A Colden; Sushant Kumar; Bolormaa Munkhbileg; Daria V Babushok
Journal:  Front Immunol       Date:  2022-01-28       Impact factor: 8.786

8.  Prediction and validation of hematopoietic stem and progenitor cell off-target editing in transplanted rhesus macaques.

Authors:  Aisha A AlJanahi; Cicera R Lazzarotto; Shirley Chen; Tae-Hoon Shin; Stefan Cordes; Xing Fan; Isabel Jabara; Yifan Zhou; David J Young; Byung-Chul Lee; Kyung-Rok Yu; Yuesheng Li; Bradley Toms; Ilker Tunc; So Gun Hong; Lauren L Truitt; Julia Klermund; Geoffroy Andrieux; Miriam Y Kim; Toni Cathomen; Saar Gill; Shengdar Q Tsai; Cynthia E Dunbar
Journal:  Mol Ther       Date:  2021-06-24       Impact factor: 11.454
  8 in total

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