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TITLE: A Brazilian patient with late infantile metachromatic leukodystrophy treated with lentiviral hematopoietic stem-cell gene therapy: A report from prenatal diagnosis to early treatment  Full Text
AUTHORS: Faqueti, Larissa; Iop, Gabrielle; Lemos da Silva, Layzon Antonio; Kubaski, Francyne; L. Borges, Henrique B.; Oliveira Netto, Alice Brinckmann; Brusius Facchin, Ana C.; Leistner Segal, Sandra; Gus, Rejane; V. Sanseverino, Maria Teresa; Michelin Tirelli, Kristiane; Pasetto, Fernanda Bender; Sebastiao, Fernanda Medeiros; Silva, Thiago Oliveira; Azevedo Magalhaes, Jose Antonio; Moura de Souza, Carolina Fischinger; Recupero, Salvatore; Calbi, Valeria; Fumagalli, Francesca; Aiuti, Alessandro; Giugliani, Roberto; ...More
PUBLISHED: 2023, SOURCE: 19th Annual World Symposium in MOLECULAR GENETICS AND METABOLISM, VOLUME: 138, ISSUE: 2
INDEXED IN: WOS
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TITLE: Newborn screening of mucopolysaccharidoses: past, present, and future
AUTHORS: Nivethitha Arunkumar; Thomas J Langan; Molly Stapleton; Francyne Kubaski; Robert W Mason; Rajendra Singh; Hironori Kobayashi; Seiji Yamaguchi; Yasuyuki Suzuki; Kenji Orii; Tadao Orii; Toshiyuki Fukao; Shunji Tomatsu;
PUBLISHED: 2020, SOURCE: JOURNAL OF HUMAN GENETICS, VOLUME: 65, ISSUE: 7
INDEXED IN: WOS