Works matching IS 15524868 AND DT 2016 AND VI 172C AND IP 4

Results: 8

Angelman Syndrome: Current and Emerging Therapies in 2016.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 384, doi. 10.1002/ajmg.c.31536
By:
- Tan, Wen‐Hann;
- Bird, Lynne M.
Publication type:
Article
Gene and Cell-Based Therapies for Inherited Retinal Disorders: An Update.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 349, doi. 10.1002/ajmg.c.31534
By:
- Sengillo, Jesse D.;
- Justus, Sally;
- Tsai, Yi‐Ting;
- Cabral, Thiago;
- Tsang, Stephen H.
Publication type:
Article
Chromosome Therapy: Potential Strategies for the Correction of Severe Chromosome Aberrations.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 422, doi. 10.1002/ajmg.c.31530
By:
- Plona, Kathleen;
- Kim, Taehyun;
- Halloran, Katherine;
- Wynshaw‐Boris, Anthony
Publication type:
Article
Pharmacological and Biological Therapeutic Strategies for Osteogenesis Imperfecta.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 367, doi. 10.1002/ajmg.c.31532
By:
- Marom, Ronit;
- Lee, Yi‐Chien;
- Grafe, Ingo;
- Lee, Brendan
Publication type:
Article
Emerging Cellular and Gene Therapies for Congenital Anemias.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 332, doi. 10.1002/ajmg.c.31529
By:
- Ludwig, Leif S.;
- Khajuria, Rajiv K.;
- Sankaran, Vijay G.
Publication type:
Article
Somatic Overgrowth Disorders of the PI3K/AKT/mTOR Pathway & Therapeutic Strategies.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 402, doi. 10.1002/ajmg.c.31531
By:
- Keppler‐Noreuil, Kim M.;
- Parker, Victoria E.R.;
- Darling, Thomas N.;
- Martinez‐Agosto, Julian A.
Publication type:
Article
Treatment of Genetic Disorders—A Vision Coming Into Focus.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 311, doi. 10.1002/ajmg.c.31535
By:
- Bird, Lynne M.;
- Tan, Wen‐Hann
Publication type:
Article
Alternative Designs for Clinical Trials in Rare Diseases.
Published in:
American Journal of Medical Genetics. Part C: Seminars in Medical Genetics, 2016, v. 172C, n. 4, p. 313, doi. 10.1002/ajmg.c.31533
By:
- Abrahamyan, Lusine;
- Feldman, Brian M.;
- Tomlinson, George;
- Faughnan, Marie E.;
- Johnson, Sindhu R.;
- Diamond, Ivan R.;
- Gupta, Samir
Publication type:
Article