Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: Terminal organ weights (mg wet tissue) and body weights (grams) in female and male NPC1 −/− mice treated with either vehicle or THLs.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques:

Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: H&E stains of cerebral cortex ( A – C ), spleen ( D – F ), and liver ( G – I ) from NPC1 −/− mice or control mice at euthanasia. Panels ( A , D , G ) are NPC1 −/− mice treated with vehicle; panels ( B , E , H ) are NPC −/− mice treated with TfRMAb/pPDGFB-NPC1 THLs. Panels ( C , F , I ) are control mice. Arrows in panels ( A , D , G ) point to cholesterol-laden cells in brain, spleen, and liver, respectively. Brain sections are through cortical layers I–IV. Magnification is the same for all panels and the panel A magnification bar = 100 microns.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques: Control

Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: Electron microscopy of cerebral cortex of vehicle ( A , B ) or THL ( C , D ) treated NPC1 −/− mouse at euthanasia. Multi-lamellated vacuoles of varying size are present in the cytoplasm of brain cells. Magnification in panels ( A ) and ( C ) is the same and magnification bar in panel ( A ) is 1,000 nm; magnification in panels ( B ) and D is the same and magnification bar in panel ( B ) is 1,000 nm.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques: Electron Microscopy

Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: GFAP immunocytochemistry of thalamus of NPC1 −/− mouse treated with vehicle ( A ) or the TfRMAb/pPDGFB-NPC1 THLs ( B ). The GFAP immunocytochemistry of thalamus from a control mouse is shown in ( C ). Sections are counter-stained with hematoxylin. Magnification of all 3 panels is the same and the panel ( A ) magnification bar = 100 microns.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques: Immunocytochemistry, Control, Staining

Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: Organ concentrations (pg plasmid DNA per mg wet tissue) in female and male NPC1 −/− mice at 4 days following the last IV injection of either vehicle or THLs.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques: Plasmid Preparation, IV Injection

Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: Organ enrichment of NPC1 mRNA, relative to GAPDH mRNA, in THL treated NPC1 −/− mice as compared to vehicle treated NPC1 −/− mice.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques:

Journal: Scientific Reports

Article Title: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

doi: 10.1038/s41598-020-70290-w

Figure Lengend Snippet: NPC1 Western blot of the spleen of 3 vehicle treated NPC −/− mice and 3 THL treated NPC −/− mice. The spleens of the THL treated mice express the 200 kDa immunoreactive NPC1 protein.

Article Snippet: A human NPC1 expression plasmid (#SC120010), under the influence of the CMV promoter, is designated pCMV-NPC1, and was purchased from Origene (Rockville, MD).

Techniques: Western Blot