Detalhe da pesquisa
1.
First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B.
Mol Ther
; 30(12): 3587-3600, 2022 12 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-36299240
2.
Successful donor engraftment and repair of the blood-brain barrier in cerebral adrenoleukodystrophy.
Blood
; 133(12): 1378-1381, 2019 03 21.
Artigo
em Inglês
| MEDLINE | ID: mdl-30635285
3.
Reduced-Toxicity (BuFlu) Conditioning Is Better Tolerated but Has a Higher Second Transplantation Rate Compared to Myeloablative Conditioning (BuCy) in Children with Inherited Metabolic Disorders.
Biol Blood Marrow Transplant
; 26(3): 486-492, 2020 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-31751770
4.
Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy.
N Engl J Med
; 377(17): 1630-1638, 2017 10 26.
Artigo
em Inglês
| MEDLINE | ID: mdl-28976817
5.
Clinical trial of laronidase in Hurler syndrome after hematopoietic cell transplantation.
Pediatr Res
; 87(1): 104-111, 2020 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-31434105
6.
Rare Spontaneous Attenuation of Childhood Inflammatory Cerebral Adrenoleukodystrophy.
J Pediatr Neurol
; 18(2): 106-109, 2020 Apr.
Artigo
em Inglês
| MEDLINE | ID: mdl-32774083
7.
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).
Biol Blood Marrow Transplant
; 25(2): 328-334, 2019 02.
Artigo
em Inglês
| MEDLINE | ID: mdl-30292746
8.
Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation.
Biol Blood Marrow Transplant
; 25(3): 538-548, 2019 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-30292747
9.
Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes.
Genet Med
; 21(11): 2552-2560, 2019 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-31019279
10.
A report on state-wide implementation of newborn screening for X-linked Adrenoleukodystrophy.
Am J Med Genet A
; 179(7): 1205-1213, 2019 07.
Artigo
em Inglês
| MEDLINE | ID: mdl-31074578
11.
Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.
Genet Med
; 20(11): 1423-1429, 2018 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-29517765
12.
Allele-Level HLA Matching Impacts Key Outcomes Following Umbilical Cord Blood Transplantation for Inherited Metabolic Disorders.
Biol Blood Marrow Transplant
; 23(1): 119-125, 2017 01.
Artigo
em Inglês
| MEDLINE | ID: mdl-27989932
13.
Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience.
J Inherit Metab Dis
; 40(2): 271-280, 2017 03.
Artigo
em Inglês
| MEDLINE | ID: mdl-28054207
14.
The Frequency of Carpal Tunnel Syndrome in Hurler Syndrome After Peritransplant Enzyme Replacement Therapy: A Retrospective Comparison.
J Hand Surg Am
; 42(7): 573.e1-573.e8, 2017 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-28479223
15.
Impact of Graft-Recipient ABO Compatibility on Outcomes after Umbilical Cord Blood Transplant for Nonmalignant Disease.
Biol Blood Marrow Transplant
; 22(11): 2019-2024, 2016 11.
Artigo
em Inglês
| MEDLINE | ID: mdl-27496217
16.
Enzyme replacement therapy prior to haematopoietic stem cell transplantation in Mucopolysaccharidosis Type I: 10 year combined experience of 2 centres.
Mol Genet Metab
; 117(3): 373-7, 2016 Mar.
Artigo
em Inglês
| MEDLINE | ID: mdl-26832957
17.
Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.
Biol Blood Marrow Transplant
; 20(11): 1847-51, 2014 Nov.
Artigo
em Inglês
| MEDLINE | ID: mdl-25016194
18.
Treatment of hepatoblastoma with high-dose chemotherapy and stem cell rescue: the pediatric blood and marrow transplant consortium experience and review of the literature.
J Pediatr Hematol Oncol
; 36(5): 362-8, 2014 Jul.
Artigo
em Inglês
| MEDLINE | ID: mdl-24577552
19.
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
EBioMedicine
; 99: 104894, 2024 Jan.
Artigo
em Inglês
| MEDLINE | ID: mdl-38086156
20.
Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report.
Blood
; 118(7): 1971-8, 2011 Aug 18.
Artigo
em Inglês
| MEDLINE | ID: mdl-21586746