Concepedia

Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia

Bijina Balakrishnan, An Ding, Vi Nguyen, Christine DeAntonis, Paolo G.V. Martini, Kent Lai

Molecular Therapy · 2019 · 60 citations · 48 references

DOIFull text

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References

48

The Metabolic Basis of Inherited Diseases

R.K. Winkelmann · Archives of Dermatology · 1972 · 1.8K citations

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A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

Akin Akinc, Andreas Zumbuehl, Michael S. Goldberg et al. · Nature Biotechnology · 2008 · 1.2K citations

, , +16

Modified mRNA Vaccines Protect against Zika Virus Infection

Justin M. Richner, Sunny Himansu, Kimberly A. Dowd et al. · Cell · 2017 · 898 citations · Full text

, , +14

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

Staci Sabnis, E. Sathyajith Kumarasinghe, Timothy Salerno et al. · Molecular Therapy · 2018 · 794 citations · Full text

, , +16

Long‐term prognosis in galactosaemia: Results of a survey of 350 cases

Darrel Waggoner, Neil R.M. Buist, George N. Donnell · Journal of Inherited Metabolic Disease · 1989 · 532 citations

, , +4