Literature DB >> 32623069

Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions.

Francesca Ferrua1, Francesco Marangoni2, Alessandro Aiuti3, Maria Grazia Roncarolo4.   

Abstract

Entities:  

Keywords:  Gene therapy; Wiskott-Aldrich syndrome; hematopoietic stem/progenitor cell; lentiviral vector; reduced-intensity conditioning

Mesh：

Year:  2020        PMID： 32623069      PMCID： PMC7453879          DOI： 10.1016/j.jaci.2020.06.018

Source DB:  PubMed          Journal:  J Allergy Clin Immunol        ISSN： 0091-6749            Impact factor:   10.793


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  8 in total

Review 1.  Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.

Authors:  Marina Cavazzana; Frederic D Bushman; Annarita Miccio; Isabelle André-Schmutz; Emmanuelle Six
Journal:  Nat Rev Drug Discov       Date:  2019-06       Impact factor: 84.694

2.  Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome.

Authors:  Salima Hacein-Bey Abina; H Bobby Gaspar; Johanna Blondeau; Laure Caccavelli; Sabine Charrier; Karen Buckland; Capucine Picard; Emmanuelle Six; Nourredine Himoudi; Kimberly Gilmour; Anne-Marie McNicol; Havinder Hara; Jinhua Xu-Bayford; Christine Rivat; Fabien Touzot; Fulvio Mavilio; Annick Lim; Jean-Marc Treluyer; Sébastien Héritier; Francois Lefrère; Jeremy Magalon; Isabelle Pengue-Koyi; Géraldine Honnet; Stéphane Blanche; Eric A Sherman; Frances Male; Charles Berry; Nirav Malani; Frederic D Bushman; Alain Fischer; Adrian J Thrasher; Anne Galy; Marina Cavazzana
Journal:  JAMA       Date:  2015-04-21       Impact factor: 56.272

3.  Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.

Authors:  Francesco Marangoni; Marita Bosticardo; Sabine Charrier; Elena Draghici; Michela Locci; Samantha Scaramuzza; Cristina Panaroni; Maurilio Ponzoni; Francesca Sanvito; Claudio Doglioni; Marie Liabeuf; Bernard Gjata; Marie Montus; Katherine Siminovitch; Alessandro Aiuti; Luigi Naldini; Loïc Dupré; Maria Grazia Roncarolo; Anne Galy; Anna Villa
Journal:  Mol Ther       Date:  2009-03-03       Impact factor: 11.454

4.  Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult.

Authors:  Emma C Morris; Thomas Fox; Ronjon Chakraverty; Rita Tendeiro; Katie Snell; Christine Rivat; Sarah Grace; Kimberly Gilmour; Sarita Workman; Karen Buckland; Katie Butler; Ronnie Chee; Alan D Salama; Hazem Ibrahim; Havinder Hara; Cecile Duret; Fulvio Mavilio; Frances Male; Frederick D Bushman; Anne Galy; Siobhan O Burns; H Bobby Gaspar; Adrian J Thrasher
Journal:  Blood       Date:  2017-07-17       Impact factor: 22.113

Review 5.  Clinical Manifestations and Pathophysiological Mechanisms of the Wiskott-Aldrich Syndrome.

Authors:  Fabio Candotti
Journal:  J Clin Immunol       Date:  2017-10-30       Impact factor: 8.542

6.  Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study.

Authors:  Francesca Ferrua; Maria Pia Cicalese; Stefania Galimberti; Stefania Giannelli; Francesca Dionisio; Federica Barzaghi; Maddalena Migliavacca; Maria Ester Bernardo; Valeria Calbi; Andrea Angelo Assanelli; Marcella Facchini; Claudia Fossati; Elena Albertazzi; Samantha Scaramuzza; Immacolata Brigida; Serena Scala; Luca Basso-Ricci; Roberta Pajno; Miriam Casiraghi; Daniele Canarutto; Federica Andrea Salerio; Michael H Albert; Antonella Bartoli; Hermann M Wolf; Rossana Fiori; Paolo Silvani; Salvatore Gattillo; Anna Villa; Luca Biasco; Christopher Dott; Emily J Culme-Seymour; Koenraad van Rossem; Gillian Atkinson; Maria Grazia Valsecchi; Maria Grazia Roncarolo; Fabio Ciceri; Luigi Naldini; Alessandro Aiuti
Journal:  Lancet Haematol       Date:  2019-04-10       Impact factor: 18.959

7.  Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome.

Authors:  Lucia Sereni; Maria Carmina Castiello; Dario Di Silvestre; Patrizia Della Valle; Chiara Brombin; Francesca Ferrua; Maria Pia Cicalese; Loris Pozzi; Maddalena Migliavacca; Maria Ester Bernardo; Claudio Pignata; Roula Farah; Lucia Dora Notarangelo; Nufar Marcus; Lorella Cattaneo; Marco Spinelli; Stefania Giannelli; Marita Bosticardo; Koen van Rossem; Armando D'Angelo; Alessandro Aiuti; Pierluigi Mauri; Anna Villa
Journal:  J Allergy Clin Immunol       Date:  2019-03-27       Impact factor: 10.793

8.  Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report.

Authors:  Lauri M Burroughs; Aleksandra Petrovic; Ruta Brazauskas; Xuerong Liu; Linda M Griffith; Hans D Ochs; Jack J Bleesing; Stephanie Edwards; Christopher C Dvorak; Sonali Chaudhury; Susan E Prockop; Ralph Quinones; Frederick D Goldman; Troy C Quigg; Shanmuganathan Chandrakasan; Angela R Smith; Suhag Parikh; Blachy J Dávila Saldaña; Monica S Thakar; Rachel Phelan; Shalini Shenoy; Lisa R Forbes; Caridad Martinez; Deepak Chellapandian; Evan Shereck; Holly K Miller; Neena Kapoor; Jessie L Barnum; Hey Chong; David C Shyr; Karin Chen; Rolla Abu-Arja; Ami J Shah; Katja G Weinacht; Theodore B Moore; Avni Joshi; Kenneth B DeSantes; Alfred P Gillio; Geoffrey D E Cuvelier; Michael D Keller; Jacob Rozmus; Troy Torgerson; Michael A Pulsipher; Elie Haddad; Kathleen E Sullivan; Brent R Logan; Donald B Kohn; Jennifer M Puck; Luigi D Notarangelo; Sung-Yun Pai; David J Rawlings; Morton J Cowan
Journal:  Blood       Date:  2020-06-04       Impact factor: 25.476
  8 in total
  8 in total

Review 1.  Conditioning regimens for inborn errors of immunity: current perspectives and future strategies.

Authors:  Akira Nishimura; Satoshi Miyamoto; Kohsuke Imai; Tomohiro Morio
Journal:  Int J Hematol       Date:  2022-06-08       Impact factor: 2.490

Review 2.  Autoimmunity in Primary Immunodeficiencies (PID).

Authors:  Grace T Padron; Vivian P Hernandez-Trujillo
Journal:  Clin Rev Allergy Immunol       Date:  2022-06-01       Impact factor: 8.667

Review 3.  Inborn errors of immunity with atopic phenotypes: A practical guide for allergists.

Authors:  Riccardo Castagnoli; Vassilios Lougaris; Giuliana Giardino; Stefano Volpi; Lucia Leonardi; Francesco La Torre; Silvia Federici; Stefania Corrente; Bianca Laura Cinicola; Annarosa Soresina; Caterina Cancrini; Gian Luigi Marseglia; Fabio Cardinale
Journal:  World Allergy Organ J       Date:  2021-02-22       Impact factor: 4.084

4.  Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report.

Authors:  Sabina Cenciarelli; Valeria Calbi; Federica Barzaghi; Maria Ester Bernardo; Chiara Oltolini; Maddalena Migliavacca; Vera Gallo; Francesca Tucci; Federico Fraschetta; Elena Albertazzi; Elena Sophia Fratini; Giulia Consiglieri; Stefania Giannelli; Francesca Dionisio; Claudia Sartirana; Sara Racca; Chiara Camesasca; Giovanni Peretto; Rita Daverio; Antonio Esposito; Francesco De Cobelli; Paolo Silvani; Marco Rabusin; Andrea Cara; Daria Trabattoni; Stefania Dispinseri; Gabriella Scarlatti; Lorenzo Piemonti; Vito Lampasona; Maria Pia Cicalese; Alessandro Aiuti; Francesca Ferrua
Journal:  Front Immunol       Date:  2020-11-24       Impact factor: 7.561

Review 5.  Gene Therapy for Primary Immunodeficiency.

Authors:  Benjamin C Houghton; Claire Booth
Journal:  Hemasphere       Date:  2020-12-29

Review 6.  Gene Therapies for Primary Immune Deficiencies.

Authors:  Lisa A Kohn; Donald B Kohn
Journal:  Front Immunol       Date:  2021-02-25       Impact factor: 7.561

7.  Gene therapy for Whiskott-Aldrich syndrome: The latest news.

Authors:  Marina Cavazzana; Adrian Thrasher
Journal:  Clin Transl Med       Date:  2022-04

Review 8.  Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases.

Authors:  Francesca Tucci; Samantha Scaramuzza; Alessandro Aiuti; Alessandra Mortellaro
Journal:  Mol Ther       Date:  2020-11-20       Impact factor: 11.454
  8 in total

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