Last Posted: Feb 24, 2019
- Gene therapy first to 'halt' most common cause of blindness
F Walsh, BBC News, February 18, 2019 - Gene therapy was a ‘last shot’
Harvard Gazette, February 2019 - Cystic fibrosis - an example of personalized and precision medicine.
Skov Marianne et al. APMIS : acta pathologica, microbiologica, et immunologica Scandinavica 2019 Feb - Inborn errors of metabolism in the 21 st century: past to present.
Arnold Georgianne L et al. Annals of translational medicine 2018 Dec 6(24) 467 - The location of exon 4 mutations in RP1 raises challenges for genetic counselling and gene therapy.
Nanda A et al. American journal of ophthalmology 2019 Feb - Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges.
Ghiaccio Valentina et al. Molecular diagnosis & therapy 2019 Jan - Preventive medicine can be more precise and precision medicine can be more preventive!
MJ Khoury, CDC Blog, January 29, 2019 - The 1st WFH Gene Therapy Round Table: Understanding the landscape and challenges of gene therapy for haemophilia around the world.
Pierce Glenn F et al. Haemophilia : the official journal of the World Federation of Hemophilia 2019 Jan - Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic?
Cornel Martina C et al. European journal of human genetics : EJHG 2018 Dec - Moving towards a cure in genetics: what is needed to bring somatic gene therapy to the clinic
MC Cornel et al, Eur J Hum Genetics, December 19, 2018
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