Phase 1 trial of an RNA interference therapy for acute intermittent porphyria

  • Eliane Sardh
  • , Pauline Harper
  • , Manisha Balwani
  • , Penelope Stein
  • , David Rees
  • , D. Montgomery Bissell
  • , Robert Desnick
  • , Charles Parker
  • , John Phillips
  • , Herbert L. Bonkovsky
  • , Daphne Vassiliou
  • , Craig Penz
  • , Amy Chan-Daniels
  • , Qiuling He
  • , William Querbes
  • , Kevin Fitzgerald
  • , Jae B. Kim
  • , Pushkal Garg
  • , Akshay Vaishnaw
  • , Amy R. Simon
  • Karl E. Anderson

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237 Scopus citations

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Medicine and Dentistry

Biochemistry, Genetics and Molecular Biology