Last Posted: May 31, 2018
- Gene therapy is saving children’s lives—but screening to discover who needs it is lagging behind
E Mullin, Tech Review, May 31, 2018 - Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.
Baruteau Julien et al. Journal of inherited metabolic disease 2017 Jul 40(4) 497-517 - Peptide probes detect misfolded transthyretin oligomers in plasma of hereditary amyloidosis patients.
Schonhoft Joseph D et al. Science translational medicine 2017 Sep 9(407) - FDA plans to speed path to approval for some gene therapies, starting with hemophilia
I. Swetlitz, StatNews, May 22, 2018 - Gene editing in genetic medicine
TGMI, May 18, 2018 - Global report on primary immunodeficiencies: 2018 update from the Jeffrey Modell Centers Network on disease classification, regional trends, treatment modalities, and physician reported outcomes.
Modell Vicki et al. Immunologic research 2018 May - Next-generation sequencing in drug development: target identification and genetically stratified clinical trials.
Torshizi Abolfazl Doostparast et al. Drug discovery today 2018 May - Gene therapy reduces need for transfusions in severe blood disorder
NIH Research Matters, May 1, 2018 - Gene Therapy for Chronic HBV-Can We Eliminate cccDNA?
Bloom Kristie et al. Genes 2018 Apr (4) - Gene Therapy as a Curative Option for β-Thalassemia.
Biffi Alessandra et al. The New England journal of medicine 2018 378(16) 1551-1552
.png)
No hay comentarios:
Publicar un comentario