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Timothy W. Yu

harvard medical school

58H-index
173Paper Count
2.4WCitation Count
Published Papers 72
Publication Date
Guidance on communication and informed consent with patients and their families for experimental individualized treatments
err2026-05-26
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errAnnemieke Aartsma-Rus; Claudio M. de Gusmao; Scott Demarest; Matias Wagner; Georg Marckmann; Charlotte Kogel; Roseline Favresse; Rebecca Schuele; Matthis Synofzik; Holm Graessner; Lynn Bush; Timothy W. Yu
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Paying for precision: funding approaches for N-of-1 trials of individualized gene targeted therapies
err2026-05-21
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errNicole Nolen; Annemieke Aartsma-Rus; Christine Caneva; Curtis R. Coughlin II; Margot A. Cousin; Catherine Douthwright; Holm Graessner; Olivia Kim-McManus; Ashley Kuniholm; Stefanie Leonard; Andrea Martinsen; Margaret Meserve; Matthis Synofzik; Booma Yandava; Timothy W. Yu; Scott Demarest; Roger J. Paxton
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RNA targeting therapy for a prenatally enriched potassium channel associated with severe childhood epilepsy and premature death
err2026-04-29
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errSean R. Golinski; Karla Soriano; Alex C. Briegel; Madeline C. Burke; Sheng Tang; Gemma L. Carvill; Emma Sherrill; Claudia Lentucci; Timothy W. Yu; Tojo Nakayama; Ruilong Hu; Richard S. Smith
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Antisense oligonucleotide-mediated knockdown therapy in two infants with severe KCNT1 epileptic encephalopathy
err2026-04-14
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errTojo Nakayama; Christelle M. El Achkar; Lisseth E. Burbano; Imran H. Quraishi; Jing Wu; Melody Li; Yutaro Asami; Sean R. Golinski; Emma Sherrill; Benjamin D. Goodlett; Claudio M. de Gusmao; Danielle A. Friedman; Claudia Lentucci; Victoria Suslovitch; Olivia Riccardi; Kamli N. W. Faour; Ashley Kuniholm; Aubrie Soucy Verran; Siobhan Coffman; Banu Ahtam; Boxun Zhao; Diana H. Chin; Renata L. DiDonato; Chunguang A. Hu; Edith Lopez; Sonia Hills; Snezana Maljevic; Helene Tran; Lynn W. Bush; Patricia Ellen Grant; Joseph R. Madsen; Richard S. Smith; Leonard K. Kaczmarek; Charles B. Berde; Timothy W. Yu
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Mitochondrial energetic failure underlies FLVCR1-related sensory neuropathy
err2026-02-14
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errFrancesca Bertino; Diletta Isabella Zanin Venturini; Eleonora Grasso; Joanna Kopecka; Chiara Salio; Barbara Gnutti; Ram Manohar Basnet; Stefania Bellini; Luca Mignani; Boxun Zhao; Felix Kleefeld; Andreas Hentschel; Francesca Magnani; Veronica Fiorito; Raluca Elena Abalai; Livia Metani; Anna Lucia Allocco; Sara Petrillo; Francesco De Giorgio; Giorgia Ammirata; Ettore Salsano; Davide Pareyson; Maja di Rocco; Angela Abicht; Emily McCourt; Rita Horvath; Heike Kölbel; Austin Larson; Andreas Roos; Timothy W. Yu; Dario Finazzi; Chiara Riganti; Emanuela Tolosano; Deborah Chiabrando
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Atrophin-1 antisense oligonucleotide provides robust protection from pathology in a fully humanized DRPLA model
err2025-12-31
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errVelvet L. Smith; Bereket Z. Gidi; Robert M. Bragg; Jeffrey P. Cantle; Aliza Ben-Varon; Briana Noble; Silvia Prades; Andrea Compton; Julie Greenfield; Joanna A. Korecka; Anya Gemos; Timothy Yu; Vikram Khurana; Holly B. Kordasiewicz; Hien T. Zhao; Melissa Barker-Haliski; Daniel D. Child; Jeffrey B. Carroll
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Response to the FDA’s proposed pathway for individualized genetic therapies
err2025-12-29
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PREAI
errTimothy W. Yu; Julia Vitarello; Kiran Musunuru; Rebecca C. Ahrens-Nicklas; David R. Liu; Michelle L. Mellion; Fyodor Urnov; Winston Yan; Srinivas Chunduru; David Barrett; Terence R. Flotte; Janet Woodcock
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Patient-Oriented Priorities for Pediatric Erythromelalgia: A Priority-Setting Process
err2025-11-02
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errOcay, Don Daniel; Halpin, Meghan; Ford, Ella; Keighley, Karen; Keighley, Neva; Ramsay, Nikki; Ramsay, Tayla; Sheridan, Camelia M.; Sheridan, Sarah M.; Tice, Kirsten R.; De Ranieri, Deirdre; Tham, See Wan; Brownstein, Catherine A.; Clinch, Jacqui; Davis, Dawn Marie; Donado, Carolina; D'Souza, Genevieve; Kattail, Deepa; Lobo, Kimberly; Ravetti, Danielle; Sandroni, Paola; Stinson, Jennifer N.; Walco, Gary A.; Walker, Suellen M.; Yu, Timothy W.; Berde, Charles B.
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Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: a retrospective cohort study
err2025-10-11
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PREAI
errDavid Cheerie; Marlen C. Lauffer; Logan Newton; Kimberly Amburgey; Danique Beijer; Bushra Haque; Brian T. Kalish; Margaret Meserve; Rachel Y. Oh; Amy Y. Pan; Miriam S. Reuter; Michael J. Szego; Anna Szuto; Annemieke Aartsma-Rus; Michelle M. Axford; Ashish R. Deshwar; James J. Dowling; Christian R. Marshall; Zhenya Ivakine; Matthis Synofzik; Timothy W. Yu; Gregory Costain
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Implementing customized genomic sequencing reports to empower providers in safety-net Neonatal Intensive Care Units
err2025-08-13
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PREAI
errAlissa M. D’Gama; Jessica Douglas; Sonia Hills; Monica H. Wojcik; Casie A. Genetti; Aubrie Soucy Verran; Margaret G. Parker; Timothy W. Yu; Pankaj B. Agrawal
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The N=1 Collaborative: advancing customized nucleic acid therapies through collaboration and data sharing
err2025-04-25
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errBelgrad, Jillian; McConnell, Erin; Leonard, Stef; Nolen, Nicole; Lauffer, Marlen C.; Watts, Jonathan K.; Yu, Timothy; Yan, Winston X.
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Pediatric erythromelalgia from multidisciplinary perspectives: a scoping review
err2025-01-16
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errOcay, Don Daniel; Graziano Maloney, Maria; D'Souza, Genevieve; Brownstein, Catherine A.; Clinch, Jacqui; Davis, Dawn Marie; De Ranieri, Deirdre; Donado, Carolina; Halpin, Meghan; Kattail, Deepa; Lee, Benjamin Howard; Lobo, Kimberly; Ravetti, Danielle; Sandroni, Paola; Stinson, Jennifer N.; Tham, See Wan; Walco, Gary A.; Walker, Suellen M.; Yu, Timothy W.; Berde, Charles B.
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RPL26 variants: A rare cause of Diamond-Blackfan anemia syndrome with multiple congenital anomalies at the forefront
err2024-12-01
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PREAI
errVanlerberghe, Clemence mence; Frenois, Fredericde; Smol, Thomas; Jourdain, Anne-Sophie; Escande, Fabienne; Ait-Yahya, Emilie; Aldeeri, Abdulrahman A.; Yu, Timothy W.; Cormier-Daire, Valerie; Ghoumid, Jamal; Jacob, Maureen; Newbury-Ecob, Ruth; Manouvrier, Sylvie; Platon, Jessica; Sailer, Sebastian; Brunelle, Perrine; Da Costa, Lydie; Petit, Florence
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LETTER TO THE EDITOR Response to Horta et al
err2024-11-01
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PREAI
errLai, Abbe; Soucy, Aubrie; Yang, Edward; Yu, Timothy; Poduri, Annapurna
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N-of-1 Studies in an Era of Precision Medicine
err2024-10-22
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PREAI
errAugustine, Erika F.; Yu, Timothy W.; Finkel, Richard S.
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Epigenomic and phenotypic characterization of DEGCAGS syndrome
err2024-10-19
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PREAI
errKarimi, Karim; Weis, Denisa; Aukrust, Ingvild; Hsieh, Tzung-Chien; Horackova, Marie; Paulsen, Julie; Mendoza Londono, Roberto; Dupuis, Lucie; Dickson, Megan; Lesman, Hellen; Lau, Tracy; Murphy, David; Hama Salih, Khalid; Al-Musawi, Bassam M. S.; Al-Obaidi, Ruqayah G. Y.; Rydzanicz, Malgorzata; Biela, Mateus; Santos, Mafalda Saraiva; Aldeeri, Abdulrahman; Gazda, Hanna T.; Pais, Lynn; Shril, Shirlee; Dollner, Henrik; Bartakke, Sandip; Laccone, Franco; Soltysova, Andrea; Kitzler, Thomas; Soliman, Neveen A.; Relator, Raissa; Levy, Michael A.; Kerkhof, Jennifer; Rzasa, Jessica; Houlden, Henry; Pilshofer, Gabriela V.; Jobst-Schwan, Tilman; Hildebrandt, Friedhelm; Sousa, Sergio B.; Maroofian, Reza; Yu, Timothy W.; Krawitz, Peter; Sadikovic, Bekim; Douzgou Houge, Sofia
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How to pay for individualized genetic medicines
err2024-06-19
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PREAI
errPian, Julia M. Y.; Owusu, Nana; Vitarello, Julia; Yan, Winston X.; Lo, Andrew W.; Yu, Timothy W.
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ATM-deficiency-induced microglial activation promotes neurodegeneration in ataxia-telangiectasia
err2024-01-01
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errLai, Jenny; Demirbas, Didem; Kim, Junho; Jeffries, Ailsa M.; Tolles, Allie; Park, Junseok; Chittenden, Thomas W.; Buckley, Patrick G.; Yu, Timothy W.; Lodato, Michael A.; Lee, Eunjung Alice
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Contribution and therapeutic implications of retroelement insertions in ataxia telangiectasia
err2023-11-01
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errZhao, Boxun; Nguyen, Minh A.; Woo, Sijae; Kim, Jinkuk; Yu, Timothy W.; Lee, Eunjung Alice
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A Multistakeholder Perspective on Advancing Individualized Therapeutics
err2023-09-13
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errPacanowski, Michael; Vitarello, Julia; Hyun, Insoo; Yu, Timothy; Zineh, Issam
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