Are we prepared to deliver gene-targeted therapies for rare diseases?
Publication
, Journal Article
Yu, TW; Kingsmore, SF; Green, RC; MacKenzie, T; Wasserstein, M; Caggana, M; Gold, NB; Kennedy, A; Kishnani, PS; Might, M; Brooks, PJ; Urv, TK ...
Published in: Am J Med Genet C Semin Med Genet
March 2023
The cost and time needed to conduct whole-genome sequencing (WGS) have decreased significantly in the last 20 years. At the same time, the number of conditions with a known molecular basis has steadily increased, as has the number of investigational new drug applications for novel gene-based therapeutics. The prospect of precision gene-targeted therapy for all seems in reach… or is it? Here we consider practical and strategic considerations that need to be addressed to establish a foundation for the early, effective, and equitable delivery of these treatments.
Duke Scholars
Published In
Am J Med Genet C Semin Med Genet
DOI
EISSN
1552-4876
Publication Date
March 2023
Volume
193
Issue
1
Start / End Page
7 / 12
Location
United States
Related Subject Headings
- Rare Diseases
- Humans
- Genetics & Heredity
- Genetic Therapy
- 3202 Clinical sciences
- 3105 Genetics
- 1103 Clinical Sciences
- 0604 Genetics
Citation
APA
Chicago
ICMJE
MLA
NLM
Yu, T. W., Kingsmore, S. F., Green, R. C., MacKenzie, T., Wasserstein, M., Caggana, M., … Urv, T. K. (2023). Are we prepared to deliver gene-targeted therapies for rare diseases? Am J Med Genet C Semin Med Genet, 193(1), 7–12. https://doi.org/10.1002/ajmg.c.32029
Yu, Timothy W., Stephen F. Kingsmore, Robert C. Green, Tippi MacKenzie, Melissa Wasserstein, Michele Caggana, Nina B. Gold, et al. “Are we prepared to deliver gene-targeted therapies for rare diseases?” Am J Med Genet C Semin Med Genet 193, no. 1 (March 2023): 7–12. https://doi.org/10.1002/ajmg.c.32029.
Yu TW, Kingsmore SF, Green RC, MacKenzie T, Wasserstein M, Caggana M, et al. Are we prepared to deliver gene-targeted therapies for rare diseases? Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):7–12.
Yu, Timothy W., et al. “Are we prepared to deliver gene-targeted therapies for rare diseases?” Am J Med Genet C Semin Med Genet, vol. 193, no. 1, Mar. 2023, pp. 7–12. Pubmed, doi:10.1002/ajmg.c.32029.
Yu TW, Kingsmore SF, Green RC, MacKenzie T, Wasserstein M, Caggana M, Gold NB, Kennedy A, Kishnani PS, Might M, Brooks PJ, Morris JA, Parisi MA, Urv TK. Are we prepared to deliver gene-targeted therapies for rare diseases? Am J Med Genet C Semin Med Genet. 2023 Mar;193(1):7–12.
Published In
Am J Med Genet C Semin Med Genet
DOI
EISSN
1552-4876
Publication Date
March 2023
Volume
193
Issue
1
Start / End Page
7 / 12
Location
United States
Related Subject Headings
- Rare Diseases
- Humans
- Genetics & Heredity
- Genetic Therapy
- 3202 Clinical sciences
- 3105 Genetics
- 1103 Clinical Sciences
- 0604 Genetics