相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia
H. Frangoul et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Insufficient Evidence for Autism-Specific Genes
Scott M. Myers et al.
AMERICAN JOURNAL OF HUMAN GENETICS (2020)
Evaluating drug targets through human loss-of-function genetic variation
Eric Vallabh Minikel et al.
NATURE (2020)
Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol
Kausik K. Ray et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia
Frederick J. Raal et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Evinacumab for Homozygous Familial Hypercholesterolemia
Frederick J. Raal et al.
NEW ENGLAND JOURNAL OF MEDICINE (2020)
Are drug targets with genetic support twice as likely to be approved? Revised estimates of the impact of genetic support for drug mechanisms on the probability of drug approval
Emily A. King et al.
PLOS GENETICS (2019)
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
D. Adams et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
M. D. Benson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
Stephen Russell et al.
LANCET (2017)
Human knockouts and phenotypic analysis in a cohort with a high rate of consanguinity
Danish Saleheen et al.
NATURE (2017)
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy
R. S. Finkel et al.
NEW ENGLAND JOURNAL OF MEDICINE (2017)
The support of human genetic evidence for approved drug indications
Matthew R. Nelson et al.
NATURE GENETICS (2015)
Exome Sequencing, ANGPTL3 Mutations, and Familial Combined Hypolipidemia
Kiran Musunuru et al.
NEW ENGLAND JOURNAL OF MEDICINE (2010)