Gene Therapy in Rare Genetic Disorders: Current Progress and Future Perspectives. [PDF]
Curr GenomicsKhawaja S, Ali RH, Ahmed I, Umair M.
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Immune Toxicities in AAV Gene Therapy: Overview for Clinicians. [PDF]
Int J Mol SciParajuli S, Gallagher T, Flotte TR.
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DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery. [PDF]
Mol Ther AdvMoeini P +8 more
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FRAME: Framework for Real-World Evidence Assessment to Mitigate Evidence Uncertainties for Efficacy/Effectiveness - An Evaluation of Regulatory and Health Technology Assessment Decision Making. [PDF]
Clin Pharmacol TherCandore G +12 more
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Promises past and future - Gene therapy and the actualisation of future expectations. [PDF]
Soc Sci MedHilberg E +3 more
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Correction to "Confidential and non-confidential drug rebates in Switzerland, 2013-2023: empirical analysis of drug rebates, trends over time and re-evaluations" [The Lancet Regional Health-Europe, volume 46, November 2024, 101094]. [PDF]
Lancet Reg Health EurRohner N +3 more
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Current State-Of-Play of the EU Advanced Therapy Medicinal Product (ATMP) Field, With an Emphasis on Belgian Human Cell and Tissue Products. [PDF]
Wound Repair RegenVerbeken G +13 more
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In vivo applications and toxicities of AAV-based gene therapies in rare diseases. [PDF]
Orphanet J Rare DisZhao Q, Peng H, Ma Y, Yuan H, Jiang H.
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Nusinersen: the antisense oligonucleotide at the forefront of spinal muscular atrophy treatment. [PDF]
RNA BiolSingh NN, Ottesen EW, Singh RN.
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A Rationally Designed AAV9-DM Capsid with Minimal Liver Tropism. [PDF]
CellsNabakowski ZC +3 more
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