Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2010 Aug 26;363(9):852-63.
doi: 10.1056/NEJMra0907180.

New therapeutic approaches to mendelian disorders

Affiliations
Free article
Review

New therapeutic approaches to mendelian disorders

Harry C Dietz. N Engl J Med. .
Free article
No abstract available

PubMed Disclaimer

Similar articles

Cited by

  • Engineering siRNA therapeutics: challenges and strategies.
    Ali Zaidi SS, Fatima F, Ali Zaidi SA, Zhou D, Deng W, Liu S. Ali Zaidi SS, et al. J Nanobiotechnology. 2023 Oct 18;21(1):381. doi: 10.1186/s12951-023-02147-z. J Nanobiotechnology. 2023. PMID: 37848888 Free PMC article. Review.
  • Precision Medicine in Oral Health and Diseases: A Systematic Review.
    Malcangi G, Patano A, Guglielmo M, Sardano R, Palmieri G, Di Pede C, de Ruvo E, Inchingolo AD, Mancini A, Inchingolo F, Bordea IR, Dipalma G, Inchingolo AM. Malcangi G, et al. J Pers Med. 2023 Apr 25;13(5):725. doi: 10.3390/jpm13050725. J Pers Med. 2023. PMID: 37240895 Free PMC article. Review.
  • Advancing drug discovery using the power of the human genome.
    Heilbron K, Mozaffari SV, Vacic V, Yue P, Wang W, Shi J, Jubb AM, Pitts SJ, Wang X. Heilbron K, et al. J Pathol. 2021 Jul;254(4):418-429. doi: 10.1002/path.5664. Epub 2021 Apr 9. J Pathol. 2021. PMID: 33748968 Free PMC article. Review.
  • Mutation update for the SATB2 gene.
    Zarate YA, Bosanko KA, Caffrey AR, Bernstein JA, Martin DM, Williams MS, Berry-Kravis EM, Mark PR, Manning MA, Bhambhani V, Vargas M, Seeley AH, Estrada-Veras JI, van Dooren MF, Schwab M, Vanderver A, Melis D, Alsadah A, Sadler L, Van Esch H, Callewaert B, Oostra A, Maclean J, Dentici ML, Orlando V, Lipson M, Sparagana SP, Maarup TJ, Alsters SI, Brautbar A, Kovitch E, Naidu S, Lees M, Smith DM, Turner L, Raggio V, Spangenberg L, Garcia-Miñaúr S, Roeder ER, Littlejohn RO, Grange D, Pfotenhauer J, Jones MC, Balasubramanian M, Martinez-Monseny A, Blok LS, Gavrilova R, Fish JL. Zarate YA, et al. Hum Mutat. 2019 Aug;40(8):1013-1029. doi: 10.1002/humu.23771. Epub 2019 Jun 18. Hum Mutat. 2019. PMID: 31021519 Free PMC article. Review.
  • Capitalizing on the heterogeneous effects of CFTR nonsense and frameshift variants to inform therapeutic strategy for cystic fibrosis.
    Sharma N, Evans TA, Pellicore MJ, Davis E, Aksit MA, McCague AF, Joynt AT, Lu Z, Han ST, Anzmann AF, Lam AN, Thaxton A, West N, Merlo C, Gottschalk LB, Raraigh KS, Sosnay PR, Cotton CU, Cutting GR. Sharma N, et al. PLoS Genet. 2018 Nov 16;14(11):e1007723. doi: 10.1371/journal.pgen.1007723. eCollection 2018 Nov. PLoS Genet. 2018. PMID: 30444886 Free PMC article.

LinkOut - more resources