Literature DB >> 29746676

Future of Care for Patients With Chronic Granulomatous Disease: Gene Therapy and Targeted Molecular Medicine.

Michael D Keller1, Luigi D Notarangelo2, Harry L Malech2.   

Abstract

Chronic granulomatous disease is a rare and potentially fatal disorder of neutrophil function. Beyond current medical management and hematopoietic stem cell transplantation, new methods of gene therapy that use lentiviral vectors or gene editing might extend curative therapies to patients who lack a suitable transplantation donor while eliminating the risk of graft-versus-host disease. Furthermore, new therapies focused on altering the biology of phagolysosomes might offer novel targeted treatments for inflammatory complications in patients with chronic granulomatous disease.

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Year:  2018        PMID： 29746676      PMCID： PMC5985732          DOI： 10.1093/jpids/piy011

Source DB:  PubMed          Journal:  J Pediatric Infect Dis Soc        ISSN： 2048-7193            Impact factor:   3.164


  41 in total

1.  Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease.

Authors:  H L Malech; P B Maples; N Whiting-Theobald; G F Linton; S Sekhsaria; S J Vowells; F Li; J A Miller; E DeCarlo; S M Holland; S F Leitman; C S Carter; R E Butz; E J Read; T A Fleisher; R D Schneiderman; D E Van Epps; S K Spratt; C A Maack; J A Rokovich; L K Cohen; J I Gallin
Journal:  Proc Natl Acad Sci U S A       Date:  1997-10-28       Impact factor: 11.205

2.  Complications of tumor necrosis factor-α blockade in chronic granulomatous disease-related colitis.

Authors:  Gulbu Uzel; Jordan S Orange; Nina Poliak; Beatriz E Marciano; Theo Heller; Steven M Holland
Journal:  Clin Infect Dis       Date:  2010-11-08       Impact factor: 9.079

3.  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

Review 4.  A Comprehensive Approach to the Management of Children and Adults with Chronic Granulomatous Disease.

Authors:  Isaac P Thomsen; Meaghan A Smith; Steven M Holland; C Buddy Creech
Journal:  J Allergy Clin Immunol Pract       Date:  2016-05-10

5.  Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Authors:  Marina Cavazzana-Calvo; Emmanuel Payen; Olivier Negre; Gary Wang; Kathleen Hehir; Floriane Fusil; Julian Down; Maria Denaro; Troy Brady; Karen Westerman; Resy Cavallesco; Beatrix Gillet-Legrand; Laure Caccavelli; Riccardo Sgarra; Leila Maouche-Chrétien; Françoise Bernaudin; Robert Girot; Ronald Dorazio; Geert-Jan Mulder; Axel Polack; Arthur Bank; Jean Soulier; Jérôme Larghero; Nabil Kabbara; Bruno Dalle; Bernard Gourmel; Gérard Socie; Stany Chrétien; Nathalie Cartier; Patrick Aubourg; Alain Fischer; Kenneth Cornetta; Frédéric Galacteros; Yves Beuzard; Eliane Gluckman; Frederick Bushman; Salima Hacein-Bey-Abina; Philippe Leboulch
Journal:  Nature       Date:  2010-09-16       Impact factor: 49.962

6.  PPARγ activation normalizes resolution of acute sterile inflammation in murine chronic granulomatous disease.

Authors:  Ruby Fernandez-Boyanapalli; S Courtney Frasch; David W H Riches; R William Vandivier; Peter M Henson; Donna L Bratton
Journal:  Blood       Date:  2010-08-06       Impact factor: 22.113

7.  Pioglitazone restores phagocyte mitochondrial oxidants and bactericidal capacity in chronic granulomatous disease.

Authors:  Ruby F Fernandez-Boyanapalli; S Courtney Frasch; Stacey M Thomas; Kenneth C Malcolm; Michael Nicks; Ronald J Harbeck; Claudia V Jakubzick; Raphael Nemenoff; Peter M Henson; Steven M Holland; Donna L Bratton
Journal:  J Allergy Clin Immunol       Date:  2014-12-10       Impact factor: 10.793

8.  Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease.

Authors:  Stefan Stein; Marion G Ott; Stephan Schultze-Strasser; Anna Jauch; Barbara Burwinkel; Andrea Kinner; Manfred Schmidt; Alwin Krämer; Joachim Schwäble; Hanno Glimm; Ulrike Koehl; Carolin Preiss; Claudia Ball; Hans Martin; Gudrun Göhring; Kerstin Schwarzwaelder; Wolf-Karsten Hofmann; Kadin Karakaya; Sandrine Tchatchou; Rongxi Yang; Petra Reinecke; Klaus Kühlcke; Brigitte Schlegelberger; Adrian J Thrasher; Dieter Hoelzer; Reinhard Seger; Christof von Kalle; Manuel Grez
Journal:  Nat Med       Date:  2010-01-24       Impact factor: 53.440

9.  IL-1 receptor blockade restores autophagy and reduces inflammation in chronic granulomatous disease in mice and in humans.

Authors:  Antonella de Luca; Sanne P Smeekens; Andrea Casagrande; Rossana Iannitti; Kara L Conway; Mark S Gresnigt; Jakob Begun; Theo S Plantinga; Leo A B Joosten; Jos W M van der Meer; Georgios Chamilos; Mihai G Netea; Ramnik J Xavier; Charles A Dinarello; Luigina Romani; Frank L van de Veerdonk
Journal:  Proc Natl Acad Sci U S A       Date:  2014-02-18       Impact factor: 11.205

10.  Clinical outcome in children with chronic granulomatous disease managed conservatively or with hematopoietic stem cell transplantation.

Authors:  Theresa Cole; Mark S Pearce; Andrew J Cant; Catherine M Cale; David Goldblatt; Andrew R Gennery
Journal:  J Allergy Clin Immunol       Date:  2013-07-16       Impact factor: 10.793
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  8 in total

1.  Late diagnosis of chronic granulomatous disease.

Authors:  T Barkai; R Somech; A Broides; R Gavrieli; B Wolach; N Marcus; D Hagin; T Stauber
Journal:  Clin Exp Immunol       Date:  2020-07-13       Impact factor: 4.330

2.  NOX Inhibitors: From Bench to Naxibs to Bedside.

Authors:  Mahmoud H Elbatreek; Hermann Mucke; Harald H H W Schmidt
Journal:  Handb Exp Pharmacol       Date:  2021

3.  Application of droplet digital PCR for the detection of vector copy number in clinical CAR/TCR T cell products.

Authors:  Alex Lu; Hui Liu; Rongye Shi; Yihua Cai; Jinxia Ma; Lipei Shao; Victor Rong; Nikolaos Gkitsas; Hong Lei; Steven L Highfill; Sandhya Panch; David F Stroncek; Ping Jin
Journal:  J Transl Med       Date:  2020-05-08       Impact factor: 5.531

Review 4.  Chemical Tools for Targeted Amplification of Reactive Oxygen Species in Neutrophils.

Authors:  Viktor Reshetnikov; Jonas Hahn; Christian Maueröder; Christine Czegley; Luis Enrique Munoz; Martin Herrmann; Markus H Hoffmann; Andriy Mokhir
Journal:  Front Immunol       Date:  2018-08-13       Impact factor: 7.561

Review 5.  Era of Genomic Medicine: A Narrative Review on CRISPR Technology as a Potential Therapeutic Tool for Human Diseases.

Authors:  Odatha W Kotagama; Chanika D Jayasinghe; Thelma Abeysinghe
Journal:  Biomed Res Int       Date:  2019-10-07       Impact factor: 3.411

6.  Characterization of dental pulp stem cells isolated from a patient diagnosed with Crouzon syndrome.

Authors:  Daisuke Torii; Tomoko Kobayashi; Tetsuro Horie; Takeo W Tsutsui
Journal:  J Cell Physiol       Date:  2021-01-01       Impact factor: 6.384

7.  Effects of voriconazole on population pharmacokinetics and optimization of the initial dose of tacrolimus in children with chronic granulomatous disease undergoing hematopoietic stem cell transplantation.

Authors:  Xiao Chen; Dongdong Wang; Jianger Lan; Guangfei Wang; Lin Zhu; Xiaoyong Xu; Xiaowen Zhai; Hong Xu; Zhiping Li
Journal:  Ann Transl Med       Date:  2021-09

8.  Lentiviral gene therapy rescues p47phox chronic granulomatous disease and the ability to fight Salmonella infection in mice.

Authors:  Andrea Schejtman; Walmir Cutrim Aragão-Filho; Simon Clare; Marta Zinicola; Maren Weisser; Siobhan O Burns; Claire Booth; Hubert B Gaspar; David C Thomas; Antonio Condino-Neto; Adrian J Thrasher; Giorgia Santilli
Journal:  Gene Ther       Date:  2020-06-12       Impact factor: 5.250
  8 in total

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