Publication:
Empowering Retinal Gene Therapy with a Specific Promoter for Human Rod and Cone ON-Bipolar Cells

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cris.virtual.author-orcid#PLACEHOLDER_PARENT_METADATA_VALUE#
cris.virtual.author-orcid#PLACEHOLDER_PARENT_METADATA_VALUE#
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cris.virtualsource.author-orcid35fa7fa1-f1c0-42f4-9297-3d4c60ff3e15
cris.virtualsource.author-orcidca912b60-7e51-4f59-b0d4-d623cce4fbdd
cris.virtualsource.author-orcidbd346219-cfd3-41c7-bdc5-d8cf1d8e2a74
dc.contributor.authorHulliger, Elmar Carlos
dc.contributor.authorHostettler, Simon Manuel
dc.contributor.authorKleinlogel, Sonja
dc.date.accessioned2024-10-05T09:50:26Z
dc.date.available2024-10-05T09:50:26Z
dc.date.issued2020-07-12
dc.description.abstractOptogenetic gene therapy holds promise to restore high-quality vision in blind patients and recently reached clinical trials. Although the ON-bipolar cells, the first retinal interneurons, make the most attractive targets for optogenetic vision restoration, they have remained inaccessible to human gene therapy due to the lack of a robust cell-specific promoter. We describe the design and functional evaluation of 770En_454P(hGRM6), a human GRM6 gene-derived, short promoter that drives strong and highly specific expression in both the rod- and cone-type ON-bipolar cells of the human retina. Expression also in cone-type ON-bipolar cells is of importance, since the cone-dominated macula mediates high-acuity vision and is the primary target of gene therapies. 770En_454P(hGRM6)-driven middle-wave opsin expression in ON-bipolar cells achieved lasting restoration of high visual acuity in the rd1 mouse model of late retinal degeneration. The new promoter enables precise manipulation of the inner retinal network and paves the way for clinical application of gene therapies for high-resolution optogenetic vision restoration, raising hopes of significantly improving the life quality of people suffering from blindness.
dc.description.numberOfPages15
dc.description.sponsorshipInstitut für Physiologie
dc.identifier.doi10.7892/boris.144033
dc.identifier.pmid32258214
dc.identifier.publisherDOI10.1016/j.omtm.2020.03.003
dc.identifier.urihttps://boris-portal.unibe.ch/handle/20.500.12422/54982
dc.language.isoen
dc.publisherCell Press
dc.relation.ispartofMolecular Therapy - Methods & Clinical Development
dc.relation.issn2329-0501
dc.relation.organizationDCD5A442BCD8E17DE0405C82790C4DE2
dc.subject.ddc600 - Technology::610 - Medicine & health
dc.titleEmpowering Retinal Gene Therapy with a Specific Promoter for Human Rod and Cone ON-Bipolar Cells
dc.typearticle
dspace.entity.typePublication
dspace.file.typetext
oaire.citation.endPage519
oaire.citation.startPage505
oaire.citation.volume17
oairecerif.author.affiliationInstitut für Physiologie
oairecerif.author.affiliationInstitut für Physiologie
oairecerif.author.affiliationInstitut für Physiologie
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unibe.contributor.rolecreator
unibe.contributor.rolecreator
unibe.contributor.rolecreator
unibe.date.licenseChanged2020-05-21 04:23:11
unibe.description.ispublishedpub
unibe.eprints.legacyId144033
unibe.journal.abbrevTitleMol Ther Methods Clin Dev
unibe.refereedTRUE
unibe.subtype.articlejournal

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