Musolino, Patricia L.

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

Brain endothelial dysfunction in cerebral adrenoleukodystrophy

Musolino, P. L., Gong, Y., Snyder, J. M. T., Jimenez, S., Lok, J., Lo, E. H., Moser, A. B., Grabowski, E. F., Frosch, M. P., & Eichler, F. S. (2015) · Brain

Barrier Structure and Function StudiesMetabolism and Genetic Disorders

Cited by 92

Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy

Gong, Y., Mu, D., Prabhakar, S., Moser, A., Musolino, P., Ren, J., Breakefield, X. O., Maguire, C. A., & Eichler, F. S. (2015) · Molecular Therapy

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 74

MRI brain lesions in asymptomatic boys with X-linked adrenoleukodystrophy

Liberato, A. P., Mallack, E. J., Aziz-Bose, R., Hayden, D., Lauer, A., Caruso, P. A., Musolino, P. L., & Eichler, F. S. (2019) · Neurology

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 69

Microglial dysfunction as a key pathological change in adrenomyeloneuropathy

Gong, Y., Sasidharan, N., Laheji, F., Frosch, M., Musolino, P., Tanzi, R., Kim, D. Y., Biffi, A., El Khoury, J., & Eichler, F. (2017) · Annals of Neurology

Neuroinflammation and Neurodegeneration MechanismsLysosomal Storage Disorders Research

Cited by 54

ABCD1 dysfunction alters white matter microvascular perfusion

Lauer, A., Da, X., Hansen, M. B., Boulouis, G., Ou, Y., Cai, X., Liberato Celso Pedrotti, A., Kalpathy-Cramer, J., Caruso, P., Hayden, D. L., et al. (2017) · Brain

Metabolism and Genetic DisordersBarrier Structure and Function Studies

Cited by 48

The brain penetrant PPARγ agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy

Rodríguez-Pascau, L., Vilalta, A., Cerrada, M., Traver, E., Forss-Petter, S., Weinhofer, I., Bauer, J., Kemp, S., Pina, G., Pascual, S., et al. (2021) · Science Translational Medicine

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

Cited by 40

Hypoperfusion predicts lesion progression in cerebral X-linked adrenoleukodystrophy

Musolino, P. L., Rapalino, O., Caruso, P., Caviness, V. S., & Eichler, F. S. (2012) · Brain

Metabolism and Genetic DisordersAdvanced MRI Techniques and Applications

Cited by 37

Hematopoietic stem-cell gene therapy is associated with restored white matter microvascular function in cerebral adrenoleukodystrophy

Lauer, A., Speroni, S. L., Choi, M., Da, X., Duncan, C., McCarthy, S., Krishnan, V., Lusk, C. A., Rohde, D., Hansen, M. B., et al. (2023) · Nature Communications

Virus-based gene therapy researchLysosomal Storage Disorders Research

Cited by 26