Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

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Items 201–300 of 606

1. Endocrine dysfunction in adrenoleukodystrophy
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2. Concurrent axon and myelin destruction differentiates X-linked adrenoleukodystrophy from multiple sclerosis
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4. Thirty-year clinical outcomes after haematopoietic stem cell transplantation in neuronopathic Gaucher disease
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5. Etiology and treatment of adrenoleukodystrophy: new insights from Drosophila
   Hannah B. Gordon, Lourdes Valdez, Anthea Letsou · 2018 · Disease Models & Mechanisms
6. Current avenues of gene therapy in Pompe disease
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7. Immune response of BV-2 microglial cells is impacted by peroxisomal beta-oxidation
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10. The Role of Microglia in Inherited White-Matter Disorders and Connections to Frontotemporal Dementia
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11. Presymptomatic Lesion in Childhood Cerebral Adrenoleukodystrophy
    Eric James Mallack, Keith P. van Haren, Anna C Torrey, Stephanie I. W. van de Stadt, Marc Engelen, Gerald V. Raymond, Ali S. Fatemi, Florian Eichler · 2022 · Neurology
12. Advances in HIV Gene Therapy
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13. International Society for Cell & Gene Therapy Stem Cell Engineering Committee report on the current state of hematopoietic stem and progenitor cell–based genomic therapies and the challenges faced
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14. Lentiviral Hematopoietic Stem Cell Gene Therapy Rescues Clinical Phenotypes in a Murine Model of Pompe Disease
    Giuseppa Piras, Claudia Montiel-Equihua, Yee-Ka Agnes Chan, Slawomir Wantuch, Daniel J. Stuckey, Derek G. Burke, Helen Prunty, Rahul Phadke, Darren Chambers, Armando Partida-Gaytán et al. · 2020 · Molecular Therapy — Methods & Clinical Development
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16. Recent Advancements in the Diagnosis and Treatment of Leukodystrophies
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17. The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy
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18. Sendai F/HN pseudotyped lentiviral vector transduces human ciliated and non-ciliated airway cells using α 2,3 sialylated receptors
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19. An update on the diagnosis and treatment of adrenoleukodystrophy
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20. Disorders of fatty acid homeostasis
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21. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy
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22. Gene therapy for ultrarare diseases: a geneticist’s perspective
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23. Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.
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24. Staurosporine Increases Lentiviral Vector Transduction Efficiency of Human Hematopoietic Stem and Progenitor Cells
    Gretchen Lewis, Lauryn Christiansen, Jessica McKenzie, Min Luo, Eli Pasackow, Yegor D. Smurnyy, Sean Harrington, Philip D. Gregory, Gabor Veres, Olivier Nègre et al. · 2018 · Molecular Therapy — Methods & Clinical Development
25. Current Therapeutic Approaches in Leukodystrophies: A Review
    Eliza M. Gordon‐Lipkin, Ali S. Fatemi · 2018 · Journal of Child Neurology
26. Evaluation of a panel of very long-chain lysophosphatidylcholines and acylcarnitines for screening of X-linked adrenoleukodystrophy in China
    Guo-Li Tian, Feng Xu, Kuan Jiang, Yanmin Wang, Wei Ji, Yingping Zhuang · 2020 · Clinica Chimica Acta
27. Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease
    Qiushi Liang, Eva C. Vlaar, Fabio Catalano, Joon M. Pijnenburg, Merel Stok, Yvette van Helsdingen, Arnold G. Vulto, Wendy W. J. Unger, Ans T. van der Ploeg, W.W.M. Pim Pijnappel et al. · 2022 · Molecular Therapy — Methods & Clinical Development
28. Leukodystrophies
    Amy Tara Waldman · 2018 · CONTINUUM Lifelong Learning in Neurology
29. Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease
    Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Tim Maiwald, Diana L. Clarke et al. · 2022 · Molecular Therapy — Methods & Clinical Development
30. Liver-directed gene-based therapies for inborn errors of metabolism
    Pasquale Piccolo, Alessandro Rossi, Nicola Brunetti‐Pierri · 2020 · Expert Opinion on Biological Therapy
31. X-linked Adrenoleukodystrophy: Pathology, Pathophysiology, Diagnostic Testing, Newborn Screening, and Therapies
    Bela Rui Turk, Christiane Theda, Ali S. Fatemi, Ann B. Moser · 2019 · International Journal of Developmental Neuroscience
32. Peroxisomal defects in microglial cells induce a disease-associated microglial signature
    Raas, Quentin, Tawbeh, Ali, Tahri-Joutey, Mounia, Gondcaille, Catherine, Keime, Céline, Kaiser, Romain, Trompier, Doriane, Nasser, Boubker, Leoni, Valerio, Bellanger, Emma et al. · 2023 · Frontiers in Molecular Neuroscience
33. ISAnalytics enables longitudinal and high-throughput clonal tracking studies in hematopoietic stem cell gene therapy applications
    Giulia Pais, Giulio Spinozzi, Daniela Cesana, Fabrizio Benedicenti, Alessandra Albertini, Maria Ester Bernardo, Bernhard Gentner, Eugenio Montini, Andrea Calabria · 2022 · Briefings in Bioinformatics
34. Plasma C24:0- and C26:0-lysophosphatidylcholines are reliable biomarkers for the diagnosis of peroxisomal β-oxidation disorders
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35. The role of the conditioning regimen for autologous and ex vivo genetically modified hematopoietic stem cell-based therapies: recommendations from the ISCT stem cell engineering committee
    Joseph Hai Oved, Athena L. Russell, Amy Elizabeth Dezern, Susan E. Prockop, Carmem Bonfim, Akshay Sharma, Duncan Purtill, Madhavi M. Lakkaraja, Alan Bidgoli, Senthil Velan Bhoopalan et al. · 2024 · Cytotherapy
36. Therapeutic efficacy of intracerebral hematopoietic stem cell gene therapy in an Alzheimer’s disease mouse model
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37. Allogeneic stem cell transplantation with reduced intensity conditioning for patients with adrenoleukodystrophy
    Koji Kato, Ryo Maemura, Manabu Wakamatsu, Ayako Yamamori, Motoharu Hamada, Shinsuke Kataoka, Atsushi Narita, Shunsuke Miwata, Yuko Sekiya, Nozomu Kawashima et al. · 2018 · Molecular Genetics and Metabolism Reports
38. Improvement of motor and behavioral activity in Sandhoff mice transplanted with human CD34+ cells transduced with a HexA/HexB expressing lentiviral vector
    Julie R. Beegle, Kyle J. Hendrix, Haley Maciel, Jan A. Nolta, Joseph S. Anderson · 2020 · The Journal of Gene Medicine
39. Economic impact of screening for X-linked Adrenoleukodystrophy within a newborn blood spot screening programme
    Alice Bessey, James B. Chilcott, Joanna Leaviss, Anthea Sutton · 2018 · Orphanet Journal of Rare Diseases
40. Adrenoleukodystrophy in the era of newborn screening
    Liane Eng, Molly O. Regelmann · 2019 · Current Opinion in Endocrinology Diabetes and Obesity
41. Targeted metabolomics revealed changes in phospholipids during the development of neuroinflammation in Abcd1tm1Kds mice and X‐linked adrenoleukodystrophy patients
    Matthias Kettwig, Henry Gerd Klemp, Stefan Nessler, Frank Streit, Ralph Krätzner, Hendrik Rosewich, Jutta Gärtner · 2021 · Journal of Inherited Metabolic Disease
42. Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases
    Juan Antonio Bueren, Alberto Auricchio · 2023 · Human Gene Therapy
43. Late Mortality after Allogeneic Blood or Marrow Transplantation for Inborn Errors of Metabolism: A Report from the Blood or Marrow Transplant Survivor Study-2 (BMTSS-2)
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44. Small RNAs to treat human immunodeficiency virus type 1 infection by gene therapy
    Ryan P. Goguen, Camille M.G. Malard, Robert J. Scarborough · 2019 · Current Opinion in Virology
45. Antioxidant Response in Human X-Linked Adrenoleukodystrophy Fibroblasts
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46. Recent progress in gene therapy for familial hypercholesterolemia treatment
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50. Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series
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51. A Longitudinal Analysis of Early Lesion Growth in Presymptomatic Patients with Cerebral Adrenoleukodystrophy
    Eric James Mallack, Gülce Askin, Stephanie I. W. van de Stadt, Paul A. Caruso, Patricia Leonor Musolino, Marc Engelen, Sumit N. Niogi, Florian Eichler · 2021 · American Journal of Neuroradiology
52. Identification of Two Novel Mutations of ABCD1 Gene in Pedigrees with X-Linked Adrenoleukodystrophy and Review of the Literature
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53. Ongoing production of tissue-resident macrophages from hematopoietic stem cells in healthy adult macaques
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54. Association between APOE4 and biomarkers in cerebral adrenoleukodystrophy
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55. Treatment of leukodystrophies: Advances and challenges.
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56. Pathophysiology of X-Linked Adrenoleukodystrophy: Updates on Molecular Mechanisms
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57. Practical Approach to Longitudinal Neurologic Care of Adults With X-Linked Adrenoleukodystrophy and Adrenomyeloneuropathy
    Alexandra B. Kornbluh, Aaron Baldwin, Ali S. Fatemi, Adeline L. Vanderver, Laura Ann Adang, Keith P. van Haren, Jacinda B. Sampson, Florian Eichler, Reza Sadjadi, Marc Engelen et al. · 2024 · Neurology Genetics
58. Intracerebral lentiviral ABCD1 gene therapy in an early disease onset ALD mouse model
    Jie Gong, Yunyun Liu, Tsai‐Hua Chung, Liu Xu, Troy C. Lund, Lung‐Ji Chang · 2022 · Gene Therapy
59. Barcoding of Macaque Hematopoietic Stem and Progenitor Cells: A Robust Platform to Assess Vector Genotoxicity
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60. Volume of gadolinium enhancement and successful repair of the blood-brain barrier in cerebral adrenoleukodystrophy
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67. Racial/Ethnic and Insurance Status Disparities in Distance Traveled to Access Children’s Hospital Care for Severe Illness: the Case of Children with Leukodystrophies
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68. Gene therapy: An updated overview on the promising success stories.
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