Charting a Clear Path: The ASGCT Standardized Pathways Conference

Author:

Kiem Hans-Peter,Baum Christopher,Bushman Frederic D,Byrne Barry J,Carter Barrie J,Cavagnaro Joy,Malech Harry L,Mendell Jerry R,Naldini Luigi M,Sorrentino Brian P,Williams David A,Flotte Terence R

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference16 articles.

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2. Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid alpha-glucosidase;Conlon;Hum Gene Ther,2013

3. Preclinical characterization of a recombinant adeno-associated virus type 1–pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites;Flotte;Hum Gene Ther,2007

4. Expediting drug development—the FDA's new “breakthrough therapy” designation;Sherman;N Engl J Med,2013

5. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease;Cavazzana-Calvo;Science,2000

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3. DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis;Molecular Therapy - Methods & Clinical Development;2020-06

4. Hurdles Associated with the Translational Use of Genetically Modified Cells;Current Stem Cell Reports;2018-02-17

5. Foamy Virus Vector Carries a Strong Insulator in Its Long Terminal Repeat Which Reduces Its Genotoxic Potential;Journal of Virology;2018-01

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