Sanford Children's Health Research Center

Sanford Children's Health Research Center

Sanford Children's Health Research Center logoDiseases that predominantly affect children are too often unaddressed by the biopharmaceutical industry and government funding agencies.

"I have the utmost confidence that we can discover solutions to some of the most troubling health issues that affect children."

Denny Sanford, Naming donor and Honorary Trustee

 

A world leader in providing solutions to childhood diseases

Our mission is to generate translational discoveries about such childhood diseases as diabetes, Duchenne muscular dystrophy, hypophosphatasia (bone disorders), and congenital disorders of glycosylation.

Recording

March 18, 2021

Fighting Rare Diseases

Finding Cures – Bringing Hope

Join us as we meet Hudson Freeze, Ph.D., José Luis Millán, Ph.D., and two of the patients they cured for a conversation on how their research is saving the lives of children facing rare disease.

Watch

Symposium

Feb. 25-27, 2022

Latest news

Publications

Quantitative atomic force microscopy provides new insight into matrix vesicle mineralization.

Plaut JS, Strzelecka-Kiliszek A, Bozycki L, Pikula S, Buchet R, Mebarek S, Chadli M, Bolean M, Simao AMS, Ciancaglini P, Magrini A, Rosato N, Magne D, Girard-Egrot A, Farquharson C, Esener SC, Millan JL, Bottini M

Arch Biochem Biophys 2019 May 30 ;667:14-21

Cardiac Hyaluronan Synthesis Is Critically Involved in the Cardiac Macrophage Response and Promotes Healing After Ischemia Reperfusion Injury.

Petz A, Grandoch M, Gorski DJ, Abrams M, Piroth M, Schneckmann R, Homann S, Müller J, Hartwig S, Lehr S, Yamaguchi Y, Wight TN, Gorressen S, Ding Z, Kötter S, Krüger M, Heinen A, Kelm M, Gödecke A, Flögel U, Fischer JW

Circ Res 2019 May 10 ;124(10):1433-1447

Arrest of Fetal Brain Development in ALG11-Congenital Disorder of Glycosylation.

Mulkey SB, Ng BG, Vezina GL, Bulas DI, Wolfe LA, Freeze HH, Ferreira CR

Pediatr Neurol 2019 May ;94:64-69

Active site variants in STT3A cause a dominant type I congenital disorder of glycosylation with neuromusculoskeletal findings.

Wilson MP, Garanto A, Pinto E Vairo F F, Ng BG, Ranatunga WK, Ventouratou M, Baerenfaenger M, Huijben K, Thiel C, Ashikov A, Keldermans L, Souche E, Vuillaumier-Barrot S, Dupré T, Michelakakis H, Fiumara A, Pitt J, White S, Lim SC, Gallacher L, Peters H, Rymen D, Witters P, Ribes A, Morales-Romero B, Rodríguez-Palmero A, Ballhausen D, de Lonlay P, Barone R, Janssen MCH, Jaeken J, Freeze HH, Matthijs G, Morava E, Lefeber DJ

Am J Hum Genet 2021 Oct 8 ;

The Autophagy, Inflammation and Metabolism Center international eSymposium - an early-career investigators' seminar series during the COVID-19 pandemic.

Nieto-Torres JL, Durgan J, Franco-Romero A, Grumati P, Guardia CM, Leidal AM, Mandell MA, Towers CG, Wang F

J Cell Sci 2021 Oct 1 ;134(19)

Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection.

Matsumoto T, Miyake K, Miyake N, Iijima O, Adachi K, Narisawa S, Millán JL, Orimo H, Shimada T

Mol Ther Methods Clin Dev 2021 Sep 10 ;22:330-337

Autophagy in major human diseases.

Klionsky DJ, Petroni G, Amaravadi RK, Baehrecke EH, Ballabio A, Boya P, Bravo-San Pedro JM, Cadwell K, Cecconi F, Choi AMK, Choi ME, Chu CT, Codogno P, Colombo MI, Cuervo AM, Deretic V, Dikic I, Elazar Z, Eskelinen EL, Fimia GM, Gewirtz DA, Green DR, Hansen M, Jäättelä M, Johansen T, Juhász G, Karantza V, Kraft C, Kroemer G, Ktistakis NT, Kumar S, Lopez-Otin C, Macleod KF, Madeo F, Martinez J, Meléndez A, Mizushima N, Münz C, Penninger JM, Perera RM, Piacentini M, Reggiori F, Rubinsztein DC, Ryan KM, Sadoshima J, Santambrogio L, Scorrano L, Simon HU, Simon AK, Simonsen A, Stolz A, Tavernarakis N, Tooze SA, Yoshimori T, Yuan J, Yue Z, Zhong Q, Galluzzi L, Pietrocola F

EMBO J 2021 Oct 1 ;40(19):e108863

Prenatal enzyme replacement therapy for Akp2 -/- mice with lethal hypophosphatasia.

Hasegawa A, Nakamura-Takahashi A, Kasahara M, Saso N, Narisawa S, Millán JL, Samura O, Sago H, Okamoto A, Umezawa A

Regen Ther 2021 Dec ;18:168-175

Gene Therapy Using Adeno-Associated Virus Serotype 8 Encoding TNAP-D10 Improves the Skeletal and Dentoalveolar Phenotypes in Alpl-/- Mice.

Kinoshita Y, Mohamed FF, Amadeu de Oliveira F, Narisawa S, Miyake K, Foster BL, Millán JL

J Bone Miner Res 2021 Sep ;36(9):1835-1849

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