Orchard, Paul J.

Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy

Eichler, F., Duncan, C., Musolino, P. L., Orchard, P. J., De Oliveira, S., Thrasher, A. J., Armant, M., Dansereau, C., Lund, T. C., Miller, W. P., et al. (2017) · New England Journal of Medicine

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 604

Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999

Peters, C., Charnas, L. R., Tan, Y., Ziegler, R. S., Shapiro, E. G., DeFor, T., Grewal, S. S., Orchard, P. J., Abel, S. L., Goldman, A. I., et al. (2004) · Blood

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 374

Human Osteoclast-Poor Osteopetrosis with Hypogammaglobulinemia due to TNFRSF11A (RANK) Mutations

Guerrini, M. M., Sobacchi, C., Cassani, B., Abinun, M., Kilic, S. S., Pangrazio, A., Moratto, D., Mazzolari, E., Clayton-Smith, J., Orchard, P., et al. (2008) · The American Journal of Human Genetics

Bone Metabolism and DiseasesImmunodeficiency and Autoimmune Disorders

Cited by 309

Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report

Miller, W. P., Rothman, S. M., Nascene, D., Kivisto, T., DeFor, T. E., Ziegler, R. S., Eisengart, J., Leiser, K., Raymond, G., Lund, T. C., et al. (2011) · Blood

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 290

International Recommendations for the Diagnosis and Management of Patients With Adrenoleukodystrophy

Engelen, M., van Ballegoij, W. J. C., Mallack, E. J., Van Haren, K. P., Köhler, W., Salsano, E., van Trotsenburg, A. S. P., Mochel, F., Sevin, C., Regelmann, M. O., et al. (2022) · Neurology

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 156

Survival and Functional Outcomes in Boys with Cerebral Adrenoleukodystrophy with and without Hematopoietic Stem Cell Transplantation

Raymond, G. V., Aubourg, P., Paker, A., Escolar, M., Fischer, A., Blanche, S., Baruchel, A., Dalle, J.-H., Michel, G., Prasad, V., et al. (2019) · Biology of Blood and Marrow Transplantation

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 142

Rapid Induction of Cerebral Organoids From Human Induced Pluripotent Stem Cells Using a Chemically Defined Hydrogel and Defined Cell Culture Medium

Lindborg, B. A., Brekke, J. H., Vegoe, A. L., Ulrich, C. B., Haider, K. T., Subramaniam, S., Venhuizen, S. L., Eide, C. R., Orchard, P. J., Chen, W., et al. (2016) · Stem Cells Translational Medicine

Pluripotent Stem Cells ResearchNeurogenesis and neuroplasticity mechanisms

Cited by 141

MRI surveillance of boys with X‐linked adrenoleukodystrophy identified by newborn screening: Meta‐analysis and consensus guidelines

Mallack, E. J., Turk, B. R., Yan, H., Price, C., Demetres, M., Moser, A. B., Becker, C., Hollandsworth, K., Adang, L., Vanderver, A., et al. (2021) · Journal of Inherited Metabolic Disease

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 79

A report on state-wide implementation of newborn screening for X-linked Adrenoleukodystrophy

Wiens, K., Berry, S. A., Choi, H., Gaviglio, A., Gupta, A., Hietala, A., Kenney‐Jung, D., Lund, T., Miller, W., Pierpont, E. I., et al. (2019) · American Journal of Medical Genetics Part A

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 75

Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy

Gupta, A. O., Raymond, G., Pierpont, E. I., Kemp, S., McIvor, R. S., Rayannavar, A., Miller, B., Lund, T. C., & Orchard, P. J. (2022) · Expert Opinion on Biological Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 67

Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I

Belur, L. R., Temme, A., Podetz-Pedersen, K. M., Riedl, M., Vulchanova, L., Robinson, N., Hanson, L. R., Kozarsky, K. F., Orchard, P. J., Frey, W. H., et al. (2017) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 67

Neurocognitive trajectory of boys who received a hematopoietic stem cell transplant at an early stage of childhood cerebral adrenoleukodystrophy

Pierpont, E. I., Eisengart, J. B., Shanley, R., Nascene, D., Raymond, G. V., Shapiro, E. G., Ziegler, R. S., Orchard, P. J., & Miller, W. P. (2017) · JAMA Neurology

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 62

Early diagnosis of cerebral X-linked adrenoleukodystrophy in boys with Addison’s disease improves survival and neurological outcomes

Polgreen, L. E., Chahla, S., Miller, W., Rothman, S., Tolar, J., Kivisto, T., Nascene, D., Orchard, P. J., & Petryk, A. (2011) · European Journal of Pediatrics

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 49

Neurocognitive benchmarks following transplant for emerging cerebral adrenoleukodystrophy

Pierpont, E. I., Nascene, D. R., Shanley, R., Kenney-Jung, D. L., Ziegler, R. S., Miller, W. P., Gupta, A. O., Lund, T. C., Orchard, P. J., & Eisengart, J. B. (2020) · Neurology

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 21

Volume of gadolinium enhancement and successful repair of the blood-brain barrier in cerebral adrenoleukodystrophy

Lund, T. C., Ng, M., Orchard, P. J., Loes, D. J., Raymond, G. V., Gupta, A., Kenny-Jung, D., & Nascene, D. R. (2020) · Biology of Blood and Marrow Transplantation

Lysosomal Storage Disorders ResearchMetabolism and Genetic Disorders

Cited by 12