FIGURE

Fig. 1

ID

ZDB-FIG-220701-31

Publication

Tucker et al., 2021 - Development and biological characterization of a clinical gene transfer vector for the treatment of MAK-associated retinitis pigmentosa

Other Figures

All Figure Page

Back to All Figure Page

Fig. 1

Design and generation of MAK gene transfer constructs.

A Schematic depicting the four AAV constructs generated in this study. Constructs were designed to deliver either the human canonical (MAK^CI - lacks exon 12, constructs 1 and 3) or retina-specific MAK isoform (MAK^RI - contains exon 12, constructs 2 and 4) under control of either the constitutively active CMV or EF1α promoter. B Representative image of cultured murine JK1 cells (immortalized SMA + , CD34 + testicular stromal cells, which lack endogenous expression of human MAK). Scale bar 100 μm. C, D rt-PCR analysis performed on RNA isolated from JK1 cells transduced with either AAV5-CMV-MAK^CI (Construct 1), AAV5-CMV-MAK^RI (Construct 2), AAV5-EF1α-MAK^CI (Construct 3), or AAV5-EF1α-MAK^RI (Construct 4). Each construct is capable of driving robust expression of exon-9-containing human MAK transcript (C). Unlike the MAK^CI constructs, both AAV5-CMV-MAK^RI and AAV5-EF1α-MAK^RI constructs are capable of driving expression of the exon-12-containing retina-specific MAK transcript (D). Arrows denote rt-PCR primer positions.

Expression Data

Expression Detail

Antibody Labeling

Phenotype Data

Phenotype Detail

Acknowledgments

This image is the copyrighted work of the attributed author or publisher, and ZFIN has permission only to display this image to its users. Additional permissions should be obtained from the applicable author or publisher of the image. Full text @ Gene Ther.