Literature DB >> 20493396

Hematopoietic stem cell transplantation for chronic granulomatous disease.

Reinhard A Seger1.   

Abstract

Chronic granulomatous disease (CGD) is a primary immunodeficiency disease that is caused by the lack of 1 of 5 subunits of the superoxide-producing nicotinamide adenine dinucleotide phosphate oxidase of neutrophils, macrophages, and eosinophils. Allogeneic hematopoietic stem cell transplantation (HSCT) is currently the only curative treatment for CGD and can be offered to selected patients. Improved outcome with supportive care and high clinical variability in the disease course, however, make selection of eligible patients for HSCT difficult. This article addresses recent progress in HSCT for CGD, delineates present limitations, and points to future developments. Copyright (c) 2010 Elsevier Inc. All rights reserved.

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Year:  2010        PMID: 20493396     DOI: 10.1016/j.iac.2010.01.003

Source DB:  PubMed          Journal:  Immunol Allergy Clin North Am        ISSN: 0889-8561            Impact factor:   3.479


  15 in total

1.  Cutaneous manifestations of DOCK8 deficiency syndrome.

Authors:  Emily Y Chu; Alexandra F Freeman; Huie Jing; Edward W Cowen; Joie Davis; Helen C Su; Steven M Holland; Maria L Chanco Turner
Journal:  Arch Dermatol       Date:  2011-09-19

2.  Chronic granulomatous disease.

Authors:  Lorenzo Loffredo
Journal:  Intern Emerg Med       Date:  2011-10       Impact factor: 3.397

3.  Oxidase-deficient neutrophils from X-linked chronic granulomatous disease iPS cells: functional correction by zinc finger nuclease-mediated safe harbor targeting.

Authors:  Jizhong Zou; Colin L Sweeney; Bin-Kuan Chou; Uimook Choi; Jason Pan; Hongmei Wang; Sarah N Dowey; Linzhao Cheng; Harry L Malech
Journal:  Blood       Date:  2011-03-16       Impact factor: 22.113

4.  Clinical, Immunological, and Molecular Findings of Patients with p47phox Defect Chronic Granulomatous Disease (CGD) in Indian Families.

Authors:  Manasi Kulkarni; Mukesh Desai; Maya Gupta; Aparna Dalvi; Prasad Taur; Antony Terrance; Sunil Bhat; Mamta Manglani; Revathi Raj; Ira Shah; Manisha Madkaikar
Journal:  J Clin Immunol       Date:  2016-10-03       Impact factor: 8.317

5.  Chronic Granulomatous Disease-Associated IBD Resolves and Does Not Adversely Impact Survival Following Allogeneic HCT.

Authors:  Rebecca A Marsh; Jennifer W Leiding; Brent R Logan; Linda M Griffith; Danielle E Arnold; Elie Haddad; E Liana Falcone; Ziyan Yin; Kadam Patel; Erin Arbuckle; Jack J Bleesing; Kathleen E Sullivan; Jennifer Heimall; Lauri M Burroughs; Suzanne Skoda-Smith; Shanmuganathan Chandrakasan; Lolie C Yu; Benjamin R Oshrine; Geoffrey D E Cuvelier; Monica S Thakar; Karin Chen; Pierre Teira; Shalini Shenoy; Rachel Phelan; Lisa R Forbes; Deepak Chellapandian; Blachy J Dávila Saldaña; Ami J Shah; Katja G Weinacht; Avni Joshi; Farid Boulad; Troy C Quigg; Christopher C Dvorak; Debi Grossman; Troy Torgerson; Pamela Graham; Vinod Prasad; Alan Knutsen; Hey Chong; Holly Miller; M Teresa de la Morena; Kenneth DeSantes; Morton J Cowan; Luigi D Notarangelo; Donald B Kohn; Elizabeth Stenger; Sung-Yun Pai; John M Routes; Jennifer M Puck; Neena Kapoor; Michael A Pulsipher; Harry L Malech; Suhag Parikh; Elizabeth M Kang
Journal:  J Clin Immunol       Date:  2019-08-02       Impact factor: 8.317

6.  Treosulfan-based conditioning for allogeneic HSCT in children with chronic granulomatous disease: a multicenter experience.

Authors:  Beatriz Morillo-Gutierrez; Rita Beier; Kanchan Rao; Lauri Burroughs; Ansgar Schulz; Anna-Maria Ewins; Brenda Gibson; Petr Sedlacek; Ladislav Krol; Brigitte Strahm; Irina Zaidman; Krzysztof Kalwak; Julie-An Talano; Ann Woolfrey; Chris Fraser; Isabelle Meyts; Ingo Müller; Jacek Wachowiak; Maria Ester Bernardo; Paul Veys; Karl-Walter Sykora; Andrew R Gennery; Mary Slatter
Journal:  Blood       Date:  2016-05-23       Impact factor: 22.113

7.  Allogeneic haematopoietic stem cell transplantation as therapy for chronic granulomatous disease--single centre experience.

Authors:  Jolanta Goździk; Anna Pituch-Noworolska; Szymon Skoczeń; Wojciech Czogała; Anna Wędrychowicz; Jarosław Baran; Aleksandra Krasowska-Kwiecień; Oktawiusz Wiecha; Marek Zembala
Journal:  J Clin Immunol       Date:  2011-03-09       Impact factor: 8.317

8.  P67-phox (NCF2) lacking exons 11 and 12 is functionally active and leads to an extremely late diagnosis of chronic granulomatous disease (CGD).

Authors:  Joachim Roesler; Florian Segerer; Henner Morbach; Stefan Kleinert; Sebastian Thieme; Angela Rösen-Wolff; Johannes G Liese
Journal:  PLoS One       Date:  2012-04-13       Impact factor: 3.240

9.  Generation of X-CGD cells for vector evaluation from healthy donor CD34(+) HSCs by shRNA-mediated knock down of gp91(phox).

Authors:  Christian Brendel; Kerstin B Kaufmann; Anja Krattenmacher; Shweta Pahujani; Manuel Grez
Journal:  Mol Ther Methods Clin Dev       Date:  2014-08-27       Impact factor: 6.698

10.  Reduced-intensity conditioning for hematopoietic cell transplant for HLH and primary immune deficiencies.

Authors:  Carl E Allen; Rebecca Marsh; Peter Dawson; Catherine M Bollard; Shalini Shenoy; Philip Roehrs; Rabi Hanna; Lauri Burroughs; Leslie Kean; Julie-An Talano; Kirk R Schultz; Sung-Yun Pai; K Scott Baker; Jeffrey R Andolina; Elizabeth O Stenger; James Connelly; Alyssa Ramirez; Christopher Bryant; Mary Eapen; Michael A Pulsipher
Journal:  Blood       Date:  2018-07-11       Impact factor: 25.476

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