Literature DB >> 29700171

Short-hairpin RNA against aberrant HBBIVSI-110(G>A) mRNA restores β-globin levels in a novel cell model and acts as mono- and combination therapy for β-thalassemia in primary hematopoietic stem cells.

Petros Patsali1,2, Panayiota Papasavva1,3, Coralea Stephanou1,2, Soteroulla Christou4, Maria Sitarou4, Michael N Antoniou2, Carsten W Lederer5,3, Marina Kleanthous1,3.   

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Year:  2018        PMID： 29700171      PMCID： PMC6119153          DOI： 10.3324/haematol.2018.189357

Source DB:  PubMed          Journal:  Haematologica        ISSN： 0390-6078            Impact factor:   9.941


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  14 in total

1.  Suitability of small diagnostic peripheral-blood samples for cell-therapy studies.

Authors:  Coralea Stephanou; Panayiota Papasavva; Myria Zachariou; Petros Patsali; Marilena Epitropou; Petros Ladas; Ruba Al-Abdulla; Soteroulla Christou; Michael N Antoniou; Carsten W Lederer; Marina Kleanthous
Journal:  Cytotherapy       Date:  2017-02       Impact factor: 5.414

2.  Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.

Authors:  Christian Brendel; Swaroopa Guda; Raffaele Renella; Daniel E Bauer; Matthew C Canver; Young-Jo Kim; Matthew M Heeney; Denise Klatt; Jonathan Fogel; Michael D Milsom; Stuart H Orkin; Richard I Gregory; David A Williams
Journal:  J Clin Invest       Date:  2016-09-06       Impact factor: 14.808

3.  Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.

Authors:  Alexis A Thompson; Mark C Walters; Janet Kwiatkowski; John E J Rasko; Jean-Antoine Ribeil; Suradej Hongeng; Elisa Magrin; Gary J Schiller; Emmanuel Payen; Michaela Semeraro; Despina Moshous; Francois Lefrere; Hervé Puy; Philippe Bourget; Alessandra Magnani; Laure Caccavelli; Jean-Sébastien Diana; Felipe Suarez; Fabrice Monpoux; Valentine Brousse; Catherine Poirot; Chantal Brouzes; Jean-François Meritet; Corinne Pondarré; Yves Beuzard; Stany Chrétien; Thibaud Lefebvre; David T Teachey; Usanarat Anurathapan; P Joy Ho; Christof von Kalle; Morris Kletzel; Elliott Vichinsky; Sandeep Soni; Gabor Veres; Olivier Negre; Robert W Ross; David Davidson; Alexandria Petrusich; Laura Sandler; Mohammed Asmal; Olivier Hermine; Mariane De Montalembert; Salima Hacein-Bey-Abina; Stéphane Blanche; Philippe Leboulch; Marina Cavazzana
Journal:  N Engl J Med       Date:  2018-04-19       Impact factor: 91.245

4.  Humanized beta-thalassemia mouse model containing the common IVSI-110 splicing mutation.

Authors:  Jim Vadolas; Mikhail Nefedov; Hady Wardan; Sima Mansooriderakshan; Lucille Voullaire; Duangporn Jamsai; Robert Williamson; Panayiotis A Ioannou
Journal:  J Biol Chem       Date:  2006-01-18       Impact factor: 5.157

Review 5.  Genome-scale loss-of-function screening with a lentiviral RNAi library.

Authors:  David E Root; Nir Hacohen; William C Hahn; Eric S Lander; David M Sabatini
Journal:  Nat Methods       Date:  2006-09       Impact factor: 28.547

Review 6.  Transcription termination by the eukaryotic RNA polymerase III.

Authors:  Aneeshkumar G Arimbasseri; Keshab Rijal; Richard J Maraia
Journal:  Biochim Biophys Acta       Date:  2012-10-23

Review 7.  Lentiviral delivery of short hairpin RNAs.

Authors:  N Manjunath; Haoquan Wu; Sandesh Subramanya; Premlata Shankar
Journal:  Adv Drug Deliv Rev       Date:  2009-03-31       Impact factor: 15.470

8.  Correction of aberrant pre-mRNA splicing by antisense oligonucleotides in beta-thalassemia Egyptian patients with IVSI-110 mutation.

Authors:  Amal El-Beshlawy; Azza Mostafa; Ilham Youssry; Hala Gabr; Iman M Mansour; Manar El-Tablawy; Mona Aziz; Ibtissam R Hussein
Journal:  J Pediatr Hematol Oncol       Date:  2008-04       Impact factor: 1.289

9.  Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.

Authors:  Laura Breda; Carla Casu; Sara Gardenghi; Nicoletta Bianchi; Luca Cartegni; Mohandas Narla; Karina Yazdanbakhsh; Marco Musso; Deepa Manwani; Jane Little; Lawrence B Gardner; Dorothy A Kleinert; Eugenia Prus; Eitan Fibach; Robert W Grady; Patricia J Giardina; Roberto Gambari; Stefano Rivella
Journal:  PLoS One       Date:  2012-03-27       Impact factor: 3.240

10.  Measurement of lentiviral vector titre and copy number by cross-species duplex quantitative PCR.

Authors:  I Christodoulou; P Patsali; C Stephanou; M Antoniou; M Kleanthous; C W Lederer
Journal:  Gene Ther       Date:  2015-06-30       Impact factor: 5.250
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  6 in total

1.  Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.

Authors:  Petros Patsali; Giandomenico Turchiano; Panayiota Papasavva; Marianna Romito; Constantinos C Loucari; Coralea Stephanou; Soteroulla Christou; Maria Sitarou; Claudio Mussolino; Tatjana I Cornu; Michael N Antoniou; Carsten W Lederer; Toni Cathomen; Marina Kleanthous
Journal:  Haematologica       Date:  2019-04-19       Impact factor: 9.941

2.  Co-transcriptional splicing regulates 3' end cleavage during mammalian erythropoiesis.

Authors:  Kirsten A Reimer; Claudia A Mimoso; Karen Adelman; Karla M Neugebauer
Journal:  Mol Cell       Date:  2021-01-12       Impact factor: 17.970

3.  The Scope for Thalassemia Gene Therapy by Disruption of Aberrant Regulatory Elements.

Authors:  Petros Patsali; Claudio Mussolino; Petros Ladas; Argyro Floga; Annita Kolnagou; Soteroula Christou; Maria Sitarou; Michael N Antoniou; Toni Cathomen; Carsten Werner Lederer; Marina Kleanthous
Journal:  J Clin Med       Date:  2019-11-13       Impact factor: 4.241

4.  Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia.

Authors:  Tiwaporn Nualkaew; Karine Sii-Felice; Marie Giorgi; Bradley McColl; Julie Gouzil; Astrid Glaser; Hsiao P J Voon; Hsin Y Tee; George Grigoriadis; Saovaros Svasti; Suthat Fucharoen; Suradej Hongeng; Philippe Leboulch; Emmanuel Payen; Jim Vadolas
Journal:  Mol Ther       Date:  2021-05-01       Impact factor: 12.910

5.  CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation.

Authors:  Panayiota L Papasavva; Petros Patsali; Constantinos C Loucari; Ryo Kurita; Yukio Nakamura; Marina Kleanthous; Carsten W Lederer
Journal:  Int J Mol Sci       Date:  2022-01-19       Impact factor: 5.923

6.  Relative and Absolute Quantification of Aberrant and Normal Splice Variants in HBBIVSI-110 (G > A) β-Thalassemia.

Authors:  Petros Patsali; Panayiota Papasavva; Soteroulla Christou; Maria Sitarou; Michael N Antoniou; Carsten W Lederer; Marina Kleanthous
Journal:  Int J Mol Sci       Date:  2020-09-11       Impact factor: 5.923
  6 in total

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