Eichler, Florian

X-linked adrenoleukodystrophy in a chimpanzee due to an ABCD1 mutation reported in multiple unrelated humans

Curiel, J., Steinberg, S. J., Bright, S., Snowden, A., Moser, A. B., Eichler, F., Dubbs, H. A., Hacia, J. G., Ely, J. J., Bezner, J., et al. (2017) · Molecular Genetics and Metabolism

Metabolism and Genetic DisordersLysosomal Storage Disorders Research

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An in vitro and in vivo efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs

Vasireddy, V., Maguire, C. A., Anderson, D. W., Ng, C., Gong, Y., Eichler, F., Fourcade, S., Guilera, C., Onieva, A., Sanchez, A., et al. (2024) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchLysosomal Storage Disorders Research

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Diagnostic yield of genome sequencing in children with progressive movement disorders

Schierbaum, L., Gonzalez Saez-Diez, E., Tam, A., Rong, J., Zubair, U., Bernardi, K., Yang, K., Quiroz, V., Zaman, Z., Saffari, A., et al. (2026) · Brain

Genomics and Rare DiseasesHereditary Neurological Disorders

Cited by 3