Whole‐genome sequencing holds the key to the success of gene‐targeted therapies

Author:

Vockley Jerry12ORCID,Aartsma‐Rus Annemieke3ORCID,Cohen Jennifer L.4ORCID,Cowsert Lex M.5,Howell R. Rodney6ORCID,Yu Timothy W.7ORCID,Wasserstein Melissa P.8ORCID,Defay Thomas9ORCID

Affiliation:

1. Department of Pediatrics University of Pittsburgh School of Medicine Pittsburgh Pennsylvania USA

2. Department of Human Genetics University of Pittsburgh School of Public Health Pittsburgh Pennsylvania USA

3. Leiden University Medical Center Leiden The Netherlands

4. Department of Pediatrics Duke University Medical Center Durham North Carolina USA

5. National Phenylketonuria Alliance Eau Claire Wisconsin USA

6. Miller School of Medicine University of Miami Miami Florida USA

7. Department of Pediatrics Harvard Medical School Boston Massachusetts USA

8. Department of Pediatrics Albert Einstein College of Medicine and the Children's Hospital at Montefiore Bronx New York USA

9. Alexion AstraZeneca Rare Diseases Boston Massachusetts USA

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Cited by 6 articles. 订阅此论文施引文献 订阅此论文施引文献,注册后可以免费订阅5篇论文的施引文献,订阅后可以查看论文全部施引文献

1. Current Status of Next-Generation Sequencing in Bone Genetic Diseases;International Journal of Molecular Sciences;2023-09-07

2. A framework for individualized splice-switching oligonucleotide therapy;Nature;2023-07-12

3. Note from the editors;American Journal of Medical Genetics Part C: Seminars in Medical Genetics;2023-03

4. Implications of Genomic Newborn Screening for Infant Mortality;International Journal of Neonatal Screening;2023-02-28

5. Moving away from one disease at a time: Screening, trial design, and regulatory implications of novel platform technologies;American Journal of Medical Genetics Part C: Seminars in Medical Genetics;2023-02-04

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