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Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy Borges, Beltran; Brown, Stephen M.; Chen, Wan-Jin; Clarke, Maria T.; Herzeg, Akos; Park, Jae Hong; Ross, Joshua; Kong, Lingling; Denton, Madeline; Smith, Amy K.; Lum, Tony; Zada, Fareha Moulana; Cordero, Marco; Gupta, Nalin; Cook, Sarah E.; Murray, Heather; Matson, John; Klein, Stephanie; Bennett, C. Frank; Krainer, Adrian R.; MacKenzie, Tippi C.; Sumner, Charlotte J. Share Save
Mucopolysaccharidosis type VII (MPS VII): A novel, online GUSB gene variant database Daugherty, Sean; Hamazaki, Takashi; Hegde, Madhuri; Giuglian, Roberto; Mackenzie, Tippi C.; Dilwortha, Lisa; Kielf, Mark J.; Japalaghi, Omid Khazaie; Lianoglou, Billie R.; Marsden, Deborah; Merritt, Lawrence; Nalin, Tatiele; Pansare, Vikram; Pollard, Laura; Miller, Vanessa Rangel; Richbourg, Heather; Sparks, Teresa N.; Miller, Nicole Share Save
Interim results from a first in human phase 1 clinical trial of in utero enzyme replacement therapy for lysosomal disorders Mackenzie, Tippi C.; Lianoglou, Billie; Canepa, Emma; Herzeg, Akos; Borgas, Beltran; Cohen, Jennifer L.; Bali, Deeksha; Young, Sarah; Gonzalez-Vele, Juan; Munar, Dane; Gelb, Michael H.; Herbs, Zackary (Ari) M.; Chakraborty, Pranesh; Kishnani, Priya; Harmatz, Paul Share Save
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In utero hematopoietic stem cell transplantation for Fanconi anemia Swartzrock, Leah; Dib, Carla; Denis, Morgane; Willner, Hana; Ho, Katie; Haslett, Ethan; Han, Jian; Pan, Wenjing; Byrne-Steele, Miranda; Brown, Brittany; Krampf, Mark R.; Girsen, Anna; Blumenfeld, Yair J.; El-Sayed, Yasser Y.; Roncarolo, Maria G.; MacKenzie, Tippi C.; Czechowicz, Agnieszka D. Share Save
Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice Clarke, Maria T.; Remesal, Laura; Lentz, Lea; Tan, Danielle J.; Young, David; Thapa, Slesha; Namuduri, Shalini R.; Borges, Beltran; Kirn, Georgia; Valencia, Jasmine; Lopez, Manuel E.; Lui, Jan H.; Shiow, Lawrence R.; Dindot, Scott; Villeda, Saul; Sanders, Stephan J.; Mackenzie, Tippi C. Share Save
Measurement of glycosaminoglycans in the amniotic fluid of fetuses with mucopolysaccharidoses treated in a phase I clinical trial by in utero enzyme replacement therapy Young, Sarah; Lianoglou, Billie R.; Herzeg, Akos; Borges, Beltran; Chakraborty, Pranesh; Bali, Deeksha; Gonzalez, Juan M.; Canepa, Emma; Kishnani, Priya; Cohen, Jennifer L.; Harmatz, Paul; MacKenzie, Tippi C. Share Save
Systematic evaluation of genome sequencing for the diagnostic assessment of autism spectrum disorder and fetal structural anomalies Lowther, Chelsea; Valkanas, Elise; Giordano, Jessica L.; Wang, Harold Z.; Currall, Benjamin B.; O'Keefe, Kathryn; Pierce-Hoffman, Emma; Kurtas, Nehir E.; Whelan, Christopher W.; Hao, Stephanie P.; Weisburd, Ben; Jalili, Vahid; Fu, Jack; Wong, Isaac; Collins, Ryan L.; Zhao, Xuefang; Austin-Tse, Christina A.; Evangelista, Emily; Lemire, Gabrielle; Aggarwal, Vimla S.; Lucente, Diane; Gauthier, Laura D.; Tolonen, Charlotte; Sahakian, Nareh; Stevens, Christine; An, Joon-Yong; Dong, Shan; Norton, Mary E.; Mackenzie, Tippi C.; Devlin, Bernie; Gilmore, Kelly; Powell, Bradford C.; Brandt, Alicia; Vetrini, Francesco; Divito, Michelle; Sanders, Stephan J.; Macarthur, Daniel G.; Hodge, Jennelle C.; O'Donnell-Luria, Anne; Rehm, Heidi L.; Vora, Neeta L.; Levy, Brynn; Brand, Harrison; Wapner, Ronald J.; Talkowski, Michael E. Share Save
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease Cohen, Jennifer L.; Chakraborty, Pranesh; Fung-Kee-Fung, Karen; Schwab, Marisa E.; Bali, Deeksha; Young, Sarah P.; Gelb, Michael H.; Khaledi, Hamid; DiBattista, Alicia; Smallshaw, Stacey; Moretti, Felipe; Wong, Derek; Lacroix, Catherine; El Demellawy, Dina; Strickland, Kyle C.; Lougheed, Jane; Moon-Grady, Anita; Lianoglou, Billie R.; Harmatz, Paul; Kishnani, Priya S.; MacKenzie, Tippi C. Share Save
The impact of in utero transfusions on perinatal outcomes in patients with alpha thalassemia major: the UCSF registry Schwab, Marisa E.; Lianoglou, Billie R.; Gano, Dawn; Velez, Juan Gonzalez; Allen, Isabel E.; Arvon, Regina; Baschat, Ahmet; Bianchi, Diana W.; Bitanga, Melissa; Bourguignon, Anne; Brown, Richard N.; Chen, Bruce; Chien, May; Davis-Nelson, Shareece; de Laat, Monique W. M.; Ekwattanakit, Supachai; Gollin, Yvonne; Hirata, Greigh; Jelin, Angie; Jolley, Jennifer; Meyer, Paul; Miller, Jena; Norton, Mary E.; Ogasawara, Keith K.; Panchalee, Tachjaree; Schindewolf, Erica; Shaw, Steven W.; Stumbaugh, Tammy; Thompson, Alexis A.; Towner, Dena; Tsai, Pai-Jong Stacy; Viprakasit, Vip; Volanakis, Emmanuel; Zhang, Li; Vichinsky, Elliott; MacKenzie, Tippi C. Share Save
In Utero Enzyme-Replacement Therapy for Infantile-Onset Pompe's Disease Cohen, Jennifer L.; Chakraborty, Pranesh; Fung-Kee-Fung, Karen; Schwab, Marisa E.; Bali, Deeksha; Young, Sarah P.; Gelb, Michael H.; Khaledi, Hamid; DiBattista, Alicia; Smallshaw, Stacey; Moretti, Felipe; Wong, Derek; Lacroix, Catherine; El Demellawy, Dina; Strickland, Kyle C.; Lougheed, Jane; Moon-Grady, Anita; Lianoglou, Billie R.; Harmatz, Paul; Kishnani, Priya S.; MacKenzie, Tippi C. Share Save
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In utero enzyme replacement therapy in a fetus with infantile-onset Pompe disease Cohen, Jennifer L.; Chakraborty, Pranesh; Fung-Kee-Fung, Karen; Schwa, Marisa E.; Bali, Deeksha; Young, Sarah; Gelb, Michael H.; Khaledi, Hamid; McIntosh, Nathan; DiBattista, Alicia; Kelly, Deidre; Smallshaw, Stacey; Lianoglou, Billie; Harmatz, Paul; Kishnani, Priya S.; MacKenzie, Tippi C. Share Save
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Consensus statement for the perinatal management of patients with α thalassemia major MacKenzie, Tippi C.; Amid, Ali; Angastiniotis, Michael; Butler, Craig; Gilbert, Sandra; Keller, Roberta L.; Kharbanda, Sandhya; Gonzalez, Juan; Kirby-Allen, Melanie; Koenig, Barbara A.; Kyono, Wade; Lal, Ashutosh; Lianoglou, Billie R.; Norton, Mary E.; Ogasawara, Keith K.; Panchalee, Tachjaree; Rosner, Mara; Schwab, Marisa; Thompson, Alexis; Waye, John S.; Vichinsky, Elliott Share Save
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