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Rare Disease Translational Research Using Model Systems

Rare diseases cover a wide range of phenotypes, affecting nearly any organ system or biological function. Individually, they might affect only a few people, making them difficult to recognize, diagnose or treat by studying humans alone. This collection, launched on Rare Disease Day 2019, features articles in DMM that show how model systems reveal new gene functions, are used in preclinical studies, and enable the screening of novel and repurposed treatments for rare diseases.

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Nadia Garibaldi; Roberta Besio; Raymond Dalgleish; Simona Villani; Aileen M. Barnes; Joan C. Marini; Antonella Forlino
Doi: 10.1242/dmm.049398
Yafang Wang; Yang Liu; Shu Liu; Xiaomeng Li; Xinxin Liu; Ming Jiao; Yuqin Yang; Xueting Luo; Fenghua Wang; Xiaoling Wan; Xiaodong Sun
Doi: 10.1242/dmm.049222
Amanda Miles; Clarke Blair; Andrew Emili; Vincent Tropepe
Doi: 10.1242/dmm.048965
Robert J. Huber
Doi: 10.1242/dmm.049152
Hannah W. Y. Ng; Jennifer A. Ogbeta; Steven J. Clapcote
Doi: 10.1242/dmm.048938
Chloe M. Stanton; Amy S. Findlay; Camilla Drake; Mohammad Z. Mustafa; Philippe Gautier; Lisa McKie; Ian J. Jackson; Veronique Vitart
Doi: 10.1242/dmm.049175
Peter A. Kropp; Rosemary Bauer; Isabella Zafra; Carina Graham; Andy Golden
Doi: 10.1242/dmm.049010
Woutje M. Berdowski; Leslie E. Sanderson; Tjakko J. van Ham
Doi: 10.1242/dmm.048925
Sara Penna; Anna Villa; Valentina Capo
Doi: 10.1242/dmm.048940
Mao Mao; Tanav Popli; Marion Jeanne; Kendall Hoff; Saunak Sen; Douglas B. Gould
Doi: 10.1242/dmm.048231
Karen I. Lange; Sofia Tsiropoulou; Katarzyna Kucharska; Oliver E. Blacque
Doi: 10.1242/dmm.046631
Ian Adams; Tao Yang; Frank M. Longo; David M. Katz
Doi: 10.1242/dmm.044685
Alaa Koleilat; Joseph A. Dugdale; Trace A. Christenson; Jeffrey L. Bellah; Aaron M. Lambert; Mark A. Masino; Stephen C. Ekker; Lisa A. Schimmenti
Doi: 10.1242/dmm.043885
Lena Marie Westermann; Lutz Fleischhauer; Jonas Vogel; Zsuzsa Jenei-Lanzl; Nataniel Floriano Ludwig; Lynn Schau; Fabio Morellini; Anke Baranowsky; Timur A. Yorgan; Giorgia Di Lorenzo; Michaela Schweizer; Bruna de Souza Pinheiro; Nicole Ruas Guarany; Fernanda Sperb-Ludwig; Fernanda Visioli; Thiago Oliveira Silva; Jamie Soul; Gretl Hendrickx; J. Simon Wiegert; Ida V. D. Schwartz; Hauke Clausen-Schaumann; Frank Zaucke; Thorsten Schinke; Sandra Pohl; Tatyana Danyukova
Doi: 10.1242/dmm.046425
Éva Saskői; Zoltán Hujber; Gábor Nyírő; István Likó; Barbara Mátyási; Gábor Petővári; Katalin Mészáros; Attila L. Kovács; László Patthy; Shreyas Supekar; Hao Fan; Gergely Sváb; László Tretter; Arunabh Sarkar; Aamir Nazir; Anna Sebestyén; Attila Patócs; Anil Mehta; Krisztina Takács-Vellai
Doi: 10.1242/dmm.044925
Arun-Kumar Kaliya-Perumal; Tom J. Carney; Philip W. Ingham
Doi: 10.1242/dmm.046441
Roberta Resaz; Davide Cangelosi; Martina Morini; Daniela Segalerba; Luca Mastracci; Federica Grillo; Maria Carla Bosco; Cristina Bottino; Irma Colombo; Alessandra Eva
Doi: 10.1242/dmm.043364
Sukalp Muzumdar; Michael Koch; Hayley Hiebert; Andreas Bapst; Alessia Gravina; Wilhelm Bloch; Hans-Dietmar Beer; Sabine Werner; Matthias Schäfer
Doi: 10.1242/dmm.042648
Sofia de Oliveira; Ruth A. Houseright; Benjamin G. Korte; Anna Huttenlocher
Doi: 10.1242/dmm.042564
Meagan S. Siehr; Cory A. Massey; Jeffrey L. Noebels
Doi: 10.1242/dmm.042515
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