Literature DB >> 22549801

The state of gene therapies: the FDA perspective.

Daniel Takefman, Wilson Bryan.   

Abstract

Mesh:

Year:  2012        PMID: 22549801      PMCID: PMC3345996          DOI: 10.1038/mt.2012.51

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   11.454


× No keyword cloud information.
  3 in total

1.  Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer.

Authors:  Aaron S Kesselheim; Jessica A Myers; Jerry Avorn
Journal:  JAMA       Date:  2011-06-08       Impact factor: 56.272

2.  LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1.

Authors:  S Hacein-Bey-Abina; C Von Kalle; M Schmidt; M P McCormack; N Wulffraat; P Leboulch; A Lim; C S Osborne; R Pawliuk; E Morillon; R Sorensen; A Forster; P Fraser; J I Cohen; G de Saint Basile; I Alexander; U Wintergerst; T Frebourg; A Aurias; D Stoppa-Lyonnet; S Romana; I Radford-Weiss; F Gross; F Valensi; E Delabesse; E Macintyre; F Sigaux; J Soulier; L E Leiva; M Wissler; C Prinz; T H Rabbitts; F Le Deist; A Fischer; M Cavazzana-Calvo
Journal:  Science       Date:  2003-10-17       Impact factor: 47.728

3.  Pivotal studies of orphan drugs approved for neurological diseases.

Authors:  Jun Mitsumoto; E Ray Dorsey; Christopher A Beck; Karl Kieburtz; Robert C Griggs
Journal:  Ann Neurol       Date:  2009-08       Impact factor: 10.422

  3 in total
  3 in total

1.  Guanidinium-rich, glycerol-derived oligocarbonates: a new class of cell-penetrating molecular transporters that complex, deliver, and release siRNA.

Authors:  Paul A Wender; Melanie A Huttner; Daryl Staveness; Jessica R Vargas; Adele F Xu
Journal:  Mol Pharm       Date:  2015-01-27       Impact factor: 4.939

Review 2.  Gene therapy for cancer: regulatory considerations for approval.

Authors:  S R Husain; J Han; P Au; K Shannon; R K Puri
Journal:  Cancer Gene Ther       Date:  2015-11-20       Impact factor: 5.987

3.  Peptide-Functionalized Dendrimer Nanocarriers for Targeted Microdystrophin Gene Delivery.

Authors:  Jessica Hersh; José Manuel Condor Capcha; Camila Iansen Irion; Guerline Lambert; Mauricio Noguera; Mohit Singh; Avinash Kaur; Emre Dikici; Joaquín J Jiménez; Lina A Shehadeh; Sylvia Daunert; Sapna K Deo
Journal:  Pharmaceutics       Date:  2021-12-15       Impact factor: 6.321

  3 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.