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TMB or not TMB as a biomarker: That is the question.
Phase I Study of the Investigational NEDD8-Activating Enzyme Inhibitor Pevonedistat (TAK-924/MLN4924) in Patients with Advanced Solid Tumors.
Dual reporter comparative indexing of rAAV pseudotyped vectors in chimpanzee airway.
A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.
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Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.
Gruntman AM, Flotte TR. Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency. Methods Mol Biol. 2017; 1639:267-275.
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PubMed
subject areas
alpha 1-Antitrypsin
alpha 1-Antitrypsin Deficiency
Gene Editing
Genetic Therapy
Humans
Liver Diseases
Lung Diseases
authors with profiles
Alisha M Gruntman DVM, PhD
Terence R Flotte MD