Highlights do World Symposium: Principais Destaques em Doenças Lisossômicas

Referências

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2. Measurement of glycosaminoglycans in the amniotic fluid of 

   fetuses with mucopolysaccharidoses treated in a phase I clinical 

   trial by in utero enzyme replacement therapy. Sarah Younga, Billie R. Lianogloub, Akos Herzegb, Beltran Borgesb, Pranesh Chakrabortyc, Deeksha Balia, Juan M. Gonzalezb, Emma Canepab, Priya Kishnania, Jennifer L. Cohena, Paul Harmatzd, Tippi C. MacKenzieb, Duke University School of Medicine, Durham, NC, USA,  

   University of California San Francisco, San Francisco, CA, USA, Newborn Screening Ontario and CHEO, Ottawa, ON, Canada, University of California, San Francisco Benioff Children’s Hospital, Oakland, CA, USA. Disponível em: Measurement of glycosaminoglycans in the amniotic fluid of fetuses with mucopolysaccharidoses treated in a phase I clinical trial by in utero enzyme replacement therapy – ScienceDirect 

3. Genome-edited hematopoietic stem cells as a curative approach 

   for Gaucher disease type 1. uisa N. Pimentel Vera, Marc Gastou, Jessica Arozqueta-Basurto, Pasqualina Colella, Allan Feng, Edina Poletto, Natalia Gomez-Ospina, Stanford University, Stanford, CA, USA. Disponível em: Redirecting (elsevier.com).