Literature DB >> 31004018

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.

Petros Patsali1,2, Giandomenico Turchiano3,4, Panayiota Papasavva1,5, Marianna Romito3,4, Constantinos C Loucari1,5, Coralea Stephanou1,2, Soteroulla Christou6, Maria Sitarou6, Claudio Mussolino3,4, Tatjana I Cornu3,4, Michael N Antoniou2, Carsten W Lederer7,5, Toni Cathomen8,4,9, Marina Kleanthous10,5.   

Abstract

Entities:  

Year:  2019        PMID： 31004018      PMCID： PMC6821606          DOI： 10.3324/haematol.2018.215178

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


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  15 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.  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.

Authors:  Petros Patsali; Panayiota Papasavva; Coralea Stephanou; Soteroulla Christou; Maria Sitarou; Michael N Antoniou; Carsten W Lederer; Marina Kleanthous
Journal:  Haematologica       Date:  2018-04-26       Impact factor: 9.941

Review 3.  Molecular basis of β thalassemia and potential therapeutic targets.

Authors:  Swee Lay Thein
Journal:  Blood Cells Mol Dis       Date:  2017-06-20       Impact factor: 3.039

4.  DNA targeting specificity of RNA-guided Cas9 nucleases.

Authors:  Patrick D Hsu; David A Scott; Joshua A Weinstein; F Ann Ran; Silvana Konermann; Vineeta Agarwala; Yinqing Li; Eli J Fine; Xuebing Wu; Ophir Shalem; Thomas J Cradick; Luciano A Marraffini; Gang Bao; Feng Zhang
Journal:  Nat Biotechnol       Date:  2013-07-21       Impact factor: 54.908

5.  An online bioinformatics tool predicts zinc finger and TALE nuclease off-target cleavage.

Authors:  Eli J Fine; Thomas J Cradick; Charles L Zhao; Yanni Lin; Gang Bao
Journal:  Nucleic Acids Res       Date:  2013-12-30       Impact factor: 16.971

6.  Protospacer adjacent motif (PAM)-distal sequences engage CRISPR Cas9 DNA target cleavage.

Authors:  Regina Cencic; Hisashi Miura; Abba Malina; Francis Robert; Sylvain Ethier; T Martin Schmeing; Josée Dostie; Jerry Pelletier
Journal:  PLoS One       Date:  2014-10-02       Impact factor: 3.240

7.  Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies.

Authors:  Constantinos C Loucari; Petros Patsali; Thamar B van Dijk; Coralea Stephanou; Panayiota Papasavva; Maria Zanti; Ryo Kurita; Yukio Nakamura; Soteroulla Christou; Maria Sitarou; Sjaak Philipsen; Carsten W Lederer; Marina Kleanthous
Journal:  Hum Gene Ther Methods       Date:  2018-02       Impact factor: 2.396

8.  IthaGenes: an interactive database for haemoglobin variations and epidemiology.

Authors:  Petros Kountouris; Carsten W Lederer; Pavlos Fanis; Xenia Feleki; John Old; Marina Kleanthous
Journal:  PLoS One       Date:  2014-07-24       Impact factor: 3.240

9.  Genetic effects on gene expression across human tissues.

Authors:  Alexis Battle; Christopher D Brown; Barbara E Engelhardt; Stephen B Montgomery
Journal:  Nature       Date:  2017-10-11       Impact factor: 49.962

Review 10.  Methodologies for Improving HDR Efficiency.

Authors:  Mingjie Liu; Saad Rehman; Xidian Tang; Kui Gu; Qinlei Fan; Dekun Chen; Wentao Ma
Journal:  Front Genet       Date:  2019-01-07       Impact factor: 4.599
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  12 in total

Review 1.  Advances in genome editing: the technology of choice for precise and efficient β-thalassemia treatment.

Authors:  Gibran Ali; Muhammad Akram Tariq; Kamran Shahid; Fridoon Jawad Ahmad; Javed Akram
Journal:  Gene Ther       Date:  2020-04-30       Impact factor: 5.250

Review 2.  Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.

Authors:  Tolulope O Rosanwo; Daniel E Bauer
Journal:  Mol Ther       Date:  2021-10-08       Impact factor: 11.454

Review 3.  Genetic Manipulation Strategies for β-Thalassemia: A Review.

Authors:  Nur Atikah Zakaria; Rosnah Bahar; Wan Zaidah Abdullah; Abdul Aziz Mohamed Yusoff; Shaharum Shamsuddin; Ridhwan Abdul Wahab; Muhammad Farid Johan
Journal:  Front Pediatr       Date:  2022-06-15       Impact factor: 3.569

Review 4.  Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies.

Authors:  Carsten W Lederer; Lola Koniali; Tina Buerki-Thurnherr; Panayiota L Papasavva; Stefania La Grutta; Amelia Licari; Frantisek Staud; Donato Bonifazi; Marina Kleanthous
Journal:  Pharmaceutics       Date:  2022-04-05       Impact factor: 6.525

Review 5.  DNA Damage: From Threat to Treatment.

Authors:  Antonio Carusillo; Claudio Mussolino
Journal:  Cells       Date:  2020-07-10       Impact factor: 6.600

6.  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

Review 7.  Genome Editing for β-Hemoglobinopathies: Advances and Challenges.

Authors:  Giacomo Frati; Annarita Miccio
Journal:  J Clin Med       Date:  2021-01-28       Impact factor: 4.241

8.  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

9.  Gene editing and CRISPR in the clinic: current and future perspectives.

Authors:  Matthew P Hirakawa; Raga Krishnakumar; Jerilyn A Timlin; James P Carney; Kimberly S Butler
Journal:  Biosci Rep       Date:  2020-04-30       Impact factor: 3.840

10.  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
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