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Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

Carmine Spampanato, Erin Feeney, Lishu Li, Monica Cardone, Jeong‐A Lim, Fabio Annunziata, Hossein Zare, Roman Polishchuk, Rosa Puertollano, Giancarlo Parenti, Andrea Ballabio, Nina Raben

Author Affiliations

  1. Carmine Spampanato1,2,3,,
  2. Erin Feeney4,,
  3. Lishu Li4,,
  4. Monica Cardone1,,
  5. Jeong‐A Lim4,,
  6. Fabio Annunziata1,,
  7. Hossein Zare4,
  8. Roman Polishchuk1,
  9. Rosa Puertollano5,
  10. Giancarlo Parenti1,6,
  11. Andrea Ballabio (ballabio{at} *,1,2,3,6 and
  12. Nina Raben (rabenn{at} *,4
  1. 1Telethon Institute of Genetics and Medicine (TIGEM), Naples, Italy
  2. 2Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA
  3. 3Jan and Dan Duncan Neurological Research Institute, Texas Children Hospital, Houston, TX, USA
  4. 4Laboratory of Muscle Stem Cells and Gene Regulation, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD, USA
  5. 5Laboratory of Cell Biology, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD, USA
  6. 6Department of Pediatrics, Federico II University, Naples, Italy
  1. * Andrea Ballabio, Tel: +39 081 613 2207; Fax: +39 081 579 0919
    Nina Raben, Tel: +1 301 496 1474; Fax: +1 301 480 63 67
  1. Spampanato and Feeney contributed equally as the two co‐first authors.

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