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Advances in gene therapy for hematologic
Advances in gene therapy for hematologic disease and considerations for transfusion medicine As the list of regulatory agency-approved gene therapies grows, these products are now in the …
Spectrum of PAH gene mutations and genotype-phenotype
Spectrum of PAH gene mutations and genotype-phenotype correlation in patients with phenylalanine hydroxylase deficiency from Shanxi province Background: Phenylalanine hydroxylase deficiency (PAHD) is an autosomal recessive inborn …
