Last Posted: Mar 29, 2019
- First gene therapy to treat rare blood disease nears European approval
A Feurstein, Stat News, March 29, 2019
- Hope and challenge: Precision medicine in bladder cancer.
Su Hongwei et al. Cancer medicine 2019 Mar
- From clinical observations and molecular dissection to novel therapeutic strategies for primary immunodeficiency disorders.
Ochs Hans D et al. American journal of medical genetics. Part A 2018 Apr 176(4) 784-803
- National Rare Diseases Registry System of China and Related Cohort Studies: Vision and Roadmap.
Feng Shi et al. Human gene therapy 2018 29(2) 128-135
- Are UK hospital pharmacy departments ready for the rise of gene therapy medicinal products?
Stoner Nicola et al. Expert opinion on biological therapy 2018 18(8) 837-840
- Mutation-Based Therapy for Duchenne Muscular Dystrophy: Antisense Treatment Arrives in the Clinic.
McNally Elizabeth M et al. Circulation 2017 136(11) 979-981
- Practical Implications of Factor IX Gene Transfer for Individuals with Hemophilia B: A Clinical Perspective.
Miesbach Wolfgang et al. Human gene therapy. Clinical development 2018 29(2) 80-89
- University Flunk-Out to Genomics Pioneer: An Interview with George Church, PhD.
Wilson James M et al. Human gene therapy. Clinical development 2018 29(3) 118-120
- The Side Effects of Million-Dollar Drugs- Gene therapies will bring hope for patients and opportunity for investors—as long as someone is willing to pay for them
S Grant, WSJ, March 2019
- Recent trends in treatment of thalassemia.
El-Beshlawy Amal et al. Blood cells, molecules & diseases 2019 Feb