Molecular genetics and metabolism | Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF

Two-tiered universal newborn screening strategy for severe combined immunodeficiency.

Molecular genetics and metabolism

McGhee SA, Stiehm ER, Cowan M, Krogstad P, McCabe ER

A Markov model to analyze cost-effectiveness of screening for severe combined immunodeficiency (SCID).

Molecular genetics and metabolism

Chan K, Davis J, Pai SY, Bonilla FA, Puck JM, Apkon M

Cellular calibrators to quantitate T-cell receptor excision circles (TRECs) in clinical samples.

Molecular genetics and metabolism

Punwani D, Gonzalez-Espinosa D, Comeau AM, Dutra A, Pak E, Puck J

Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment.

Molecular genetics and metabolism

Shapiro EG, Nestrasil I, Rudser K, Delaney K, Kovac V, Ahmed A, Yund B, Orchard PJ, Eisengart J, Niklason GR, Raiman J, Mamak E, Cowan MJ, Bailey-Olson M, Harmatz P, Shankar SP, Cagle S, Ali N, Steiner RD, Wozniak J, Lim KO, Whitley CB

Fetal enzyme replacement and stem cell transplantation in murine Sly syndrome targeting microglia.

Molecular genetics and metabolism

Quoc-Hung Nguyen, Russell Witt, Lucas Smith, Jeremy Shea, Bowen Wang, Carlo Eikani, Saul Villeda, Tippi MacKenzie

Quantitative brain MRI morphology in severe and attenuated forms of mucopolysaccharidosis type I.

Molecular genetics and metabolism

Kovac V, Shapiro EG, Rudser KD, Mueller BA, Eisengart JB, Delaney KA, Ahmed A, King KE, Yund BD, Cowan MJ, Raiman J, Mamak EG, Harmatz PR, Shankar SP, Ali N, Cagle SR, Wozniak JR, Lim KO, Orchard PJ, Whitley CB, Nestrasil I

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