Projects per year
Personal profile
Expertise related to UN Sustainable Development Goals
In 2015, UN member states agreed to 17 global Sustainable Development Goals (SDGs) to end poverty, protect the planet and ensure prosperity for all. This person’s work contributes towards the following SDG(s):
Fingerprint
- 1 Similar Profiles
Collaborations and top research areas from the last five years
-
Advanced Therapies Rare Genetic Disease Fellowship
Jarnes, J. R. (PI) & Whitley, C. B. (CoI)
7/1/24 → 6/30/25
Project: Research project
-
Long-Term Follow-up of Fabry Disease Subjects who have Undergone IV Infusion with ST-920, an rAAV2/6 Human Alpha Galactosidase A Gene Therapy
Whitley, C. B. (PI), Diethelm-Okita, B. M. (CoI), Jarnes, J. R. (CoI) & Miller, B. S. (CoI)
12/4/23 → 11/30/30
Project: Research project
-
Utilizing the PS Gene Editing System to Encode a Blood-Brain Barrier-Penetrating IDUA as a Treatment for Murine Hurler Syndrome
Przybilla, M. (PI) & Whitley, C. B. (CoI)
2/1/23 → 7/31/25
Project: Research project
-
A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients
Whitley, C. B. (PI) & Miller, B. S. (CoI)
11/15/22 → 11/16/25
Project: Research project
-
Addressing the Multisystemic Impacts of Nephropathic Cystinosis in an Adult
Jarnes, J. R., Palmer, R. S. & Whitley, C. B., Mar 2025, In: Kidney International Reports. 10, 3, p. S789-S793Research output: Contribution to journal › Article › peer-review
Open Access -
Bone marrow transplantation reverses metabolic alterations in multiple sulfatase deficiency: a case series
Pillai, N. R., Liu, N., Li, X., Li, X., Ahrens-Nicklas, R., Adang, L., Eisengart, J. B., Bronken, G., Gupta, A., Lund, T. C., Whitley, C. B., Elsea, S. H. & Orchard, P. J., Dec 2025, In: Communications Medicine. 5, 1, 12.Research output: Contribution to journal › Article › peer-review
Open Access -
Detecting the Difficult: An Intronic NPC1 Variant Hiding in Plain Sight
Brown, C. G., Bower, M., Schomaker, M., Goldstein, J., Jarnes, J., Whitley, C. B. & Pillai, N. R., 2025, (Accepted/In press) In: American Journal of Medical Genetics, Part A.Research output: Contribution to journal › Article › peer-review
Open Access -
Diffusion tensor imaging with free-water correction reveals distinctions between severe and attenuated subtypes in Mucopolysaccharidosis type I
Svatkova, A., Pasternak, O., Eisengart, J. B., Rudser, K. D., Bednařík, P., Mueller, B. A., Delaney, K. A., Shapiro, E. G., Whitley, C. B. & Nestrašil, I., Jan 2025, In: Journal of Inherited Metabolic Disease. 48, 1, e12830.Research output: Contribution to journal › Article › peer-review
Open Access -
Dose-intensive therapy (DIT) for infantile Pompe disease: A pilot study
Jarnes, J. R., Pillai, N. R., Ahmed, A., Shrestha, S., Stark, M. & Whitley, C. B., Mar 2025, In: Molecular Genetics and Metabolism Reports. 42, 101179.Research output: Contribution to journal › Article › peer-review
Open Access