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Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome.
Arguello, Annie; Meisner, René; Thomsen, Elliot R; Nguyen, Hoang N; Ravi, Ritesh; Simms, Jeffrey; Lo, Iris; Speckart, Jessica; Holtzman, Julia; Gill, Thomas M; Chan, Darren; Cheng, Yuhsiang; Chiu, Chi-Lu; Dugas, Jason C; Fang, Meng; Lopez, Isabel A; Solanoy, Hilda; Tsogtbaatar, Buyankhishig; Zhu, Yuda; Bhalla, Akhil; Henne, Kirk R; Henry, Anastasia G; Delucchi, Anthony; Costanzo, Simona; Harris, Jeffrey M; Diaz, Dolores; Scearce-Levie, Kimberly; Sanchez, Pascal E.
Affiliation
  • Arguello A; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Meisner R; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Thomsen ER; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Nguyen HN; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Ravi R; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Simms J; Behavioral Core, Gladstone Institute of Neurological Disease, San Francisco, California, USA.
  • Lo I; Behavioral Core, Gladstone Institute of Neurological Disease, San Francisco, California, USA.
  • Speckart J; Behavioral Core, Gladstone Institute of Neurological Disease, San Francisco, California, USA.
  • Holtzman J; Behavioral Core, Gladstone Institute of Neurological Disease, San Francisco, California, USA.
  • Gill TM; Behavioral Core, Gladstone Institute of Neurological Disease, San Francisco, California, USA.
  • Chan D; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Cheng Y; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Chiu CL; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Dugas JC; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Fang M; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Lopez IA; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Solanoy H; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Tsogtbaatar B; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Zhu Y; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Bhalla A; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Henne KR; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Henry AG; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Delucchi A; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Costanzo S; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Harris JM; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Diaz D; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Scearce-Levie K; Denali Therapeutics Inc., South San Francisco, California, USA.
  • Sanchez PE; Denali Therapeutics Inc., South San Francisco, California, USA.
JCI Insight ; 6(19)2021 10 08.
Article in En | MEDLINE | ID: mdl-34622797
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Receptors, Transferrin / Blood-Brain Barrier / Mucopolysaccharidosis II / Transport Vesicles / Enzyme Replacement Therapy / Iduronate Sulfatase Type of study: Prognostic_studies Limits: Animals Language: En Journal: JCI Insight Year: 2021 Document type: Article Affiliation country: United States Country of publication: United States
Fulltext
Add to My VHL
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Receptors, Transferrin / Blood-Brain Barrier / Mucopolysaccharidosis II / Transport Vesicles / Enzyme Replacement Therapy / Iduronate Sulfatase Type of study: Prognostic_studies Limits: Animals Language: En Journal: JCI Insight Year: 2021 Document type: Article Affiliation country: United States Country of publication: United States