Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency ( PKD )?: Toward an expanded definition of severe PKD
Journal article published in 2022 by
Jonathan D. Schwartz 
,
Wilma Barcellini ,
Rachel F. Grace ,
Paola Bianchi ,
Alberto Zanella,
José Luis López Lorenzo ,
Julián Sevilla ,
Ami J. Shah,
Bertil Glader,
Eileen Nicoletti,
Susana Navarro Ordoñez,
José Carlos Segovia
,
Wilma Barcellini ,
Rachel F. Grace ,
Paola Bianchi ,
Alberto Zanella,
José Luis López Lorenzo ,
Julián Sevilla ,
Ami J. Shah,
Bertil Glader,
Eileen Nicoletti,
Susana Navarro Ordoñez,
José Carlos Segovia
This paper is made freely available by the publisher.
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