Last Posted: May 21, 2020
- [Recommendations for gene therapy of spinal muscular atrophy with onasemnogene abeparvovec-AVXS-101 : Consensus paper of the German representatives of the Society for Pediatric Neurology (GNP) and the German treatment centers with collaboration of the medical scientific advisory board of the German Society for Muscular Diseases (DGM)].
Ziegler Andreas et al. Der Nervenarzt 2020 May - How I manage patients with Wiskott Aldrich syndrome.
Rivers Elizabeth et al. British journal of haematology 2019 185(4) 647-655 - Prevention methods for Treacher Collins syndrome: A systematic review.
Guo Peipei et al. International journal of pediatric otorhinolaryngology 2020 Apr 134110062 - Gene therapy strategies in the treatment of hypertrophic cardiomyopathy.
Prondzynski Maksymilian et al. Pflugers Archiv : European journal of physiology 2019 471(5) 807-815 - International Triadin Knockout Syndrome Registry.
Clemens Daniel J et al. Circulation. Genomic and precision medicine 2019 12(2) e002419 - Diagnostic and Therapeutic Applications of Genomic Medicine in Progressive, Late-Onset, Nonsyndromic Sensorineural Hearing Loss.
Jimenez Joaquin E et al. Gene 2020 Apr 144677 - Recent advances in primary immunodeficiency: from molecular diagnosis to treatment.
Bucciol Giorgia et al. F1000Research 2020 9 - Variability in Genome Editing Outcomes: Challenges for Research Reproducibility and Clinical Safety.
Teboul Lydia et al. Molecular therapy : the journal of the American Society of Gene Therapy 2020 Mar - A Need for Better Understanding Is the Major Determinant for Public Perceptions of Human Gene Editing.
McCaughey Tristan et al. Human gene therapy 2019 30(1) 36-43 - Gene therapy for hearing loss.
Omichi Ryotaro et al. Human molecular genetics 2019 28(R1) R65-R79
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