Whitley, Chester B.

Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled study

Trefz, F. K., Burton, B. K., Longo, N., Casanova, M. M.-P., Gruskin, D. J., Dorenbaum, A., Kakkis, E. D., Crombez, E. A., Grange, D. K., Harmatz, P., et al. (2009) · The Journal of Pediatrics

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Dose-dependent prevention of metabolic and neurologic disease in murine MPS II by ZFN-mediated in vivo genome editing

Laoharawee, K., DeKelver, R. C., Podetz-Pedersen, K. M., Rohde, M., Sproul, S., Nguyen, H.-O., Nguyen, T., St. Martin, S. J., Ou, L., Tom, S., et al. (2018) · Molecular Therapy

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ZFN-mediated in vivo genome editing corrects murine hurler syndrome

Ou, L., DeKelver, R. C., Rohde, M., Tom, S., Radeke, R., St. Martin, S. J., Santiago, Y., Sproul, S., Przybilla, M. J., Koniar, B. L., et al. (2019) · Molecular Therapy

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A highly efficacious PS gene editing system corrects metabolic and neurological complications of mucopolysaccharidosis type I

Ou, L., Przybilla, M. J., Ahlat, O., Kim, S., Overn, P., Jarnes, J., O’Sullivan, M. G., & Whitley, C. B. (2020) · Molecular Therapy

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A novel gene editing system to treat both Tay–Sachs and sandhoff diseases

Ou, L., Przybilla, M. J., Tăbăran, A.-F., Overn, P., O’Sullivan, M. G., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S., & Whitley, C. B. (2020) · Gene Therapy

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START, a double blind, placebo-controlled pharmacogenetic test of responsiveness to sapropterin dihydrochloride in phenylketonuria patients

Utz, J. R. J., Lorentz, C. P., Markowitz, D., Rudser, K. D., Diethelm-Okita, B., Erickson, D., & Whitley, C. B. (2012) · Molecular Genetics and Metabolism

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