Literature DB >> 31731183

Generation of two compound heterozygous HGSNAT-mutated lines from healthy induced pluripotent stem cells using CRISPR/Cas9 to model Sanfilippo C syndrome.

Noelia Benetó1, Monica Cozar1, María García-Morant1, Edgar Creus-Bachiller1, Lluïsa Vilageliu1, Daniel Grinberg1, Isaac Canals2.   

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Year:  2019        PMID： 31731183     DOI： 10.1016/j.scr.2019.101616

Source DB:  PubMed          Journal:  Stem Cell Res        ISSN： 1873-5061            Impact factor:   2.020


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  2 in total

Review 1.  Lysosomal Storage Disease-Associated Neuropathy: Targeting Stable Nucleic Acid Lipid Particle (SNALP)-Formulated siRNAs to the Brain as a Therapeutic Approach.

Authors:  Maria Francisca Coutinho; Juliana Inês Santos; Liliana S Mendonça; Liliana Matos; Maria João Prata; Amália S Jurado; Maria C Pedroso de Lima; Sandra Alves
Journal:  Int J Mol Sci       Date:  2020-08-10       Impact factor: 5.923

Review 2.  Sanfilippo Syndrome: Molecular Basis, Disease Models and Therapeutic Approaches.

Authors:  Noelia Benetó; Lluïsa Vilageliu; Daniel Grinberg; Isaac Canals
Journal:  Int J Mol Sci       Date:  2020-10-22       Impact factor: 5.923
  2 in total

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