Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS

Carola J. Maturana; Jessica L. Verpeut; Thomas J. Pisano; Zahra M. Dhanerawala; Andrew Esteves; Lynn W. Enquist; Esteban A. Engel

doi:10.1016/j.omtm.2020.04.004

Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS

Carola J. Maturana, Jessica L. Verpeut, Thomas J. Pisano, Zahra M. Dhanerawala, Andrew Esteves, Lynn W. Enquist, Esteban A. Engel

Research output: Contribution to journal › Article › peer-review

14 Scopus citations

Abstract

Recombinant adeno-associated viruses (rAAVs) are used as gene therapy vectors. However, they have a limited payload capacity and lack small, long-lasting gene promoters for single-dose gene therapies. We identified small herpesvirus latency-associated promoters showing stable, pan-neuronal transgene transcription and translation in vivo for 6 months after AAV transduction.

Original language	English (US)
Pages (from-to)	843-857
Number of pages	15
Journal	Molecular Therapy - Methods and Clinical Development
Volume	17
DOIs	https://doi.org/10.1016/j.omtm.2020.04.004
State	Published - Jun 12 2020
Externally published	Yes

Keywords

AAV
Brain
CNS
Gene Delivery
Gene Promoter
Gene Therapy
LAP
LAT
Latency-Associated Promoter
Latency-Associated Transcript
Light-Sheet Microscopy
Recombinant AAV
Retro-Orbital Injection
Spinal Cord
Viral vector
iDisco+

ASJC Scopus subject areas

Molecular Medicine
Molecular Biology
Genetics

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10.1016/j.omtm.2020.04.004

Cite this

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title = "Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS",

abstract = "Recombinant adeno-associated viruses (rAAVs) are used as gene therapy vectors. However, they have a limited payload capacity and lack small, long-lasting gene promoters for single-dose gene therapies. We identified small herpesvirus latency-associated promoters showing stable, pan-neuronal transgene transcription and translation in vivo for 6 months after AAV transduction.",

keywords = "AAV, Brain, CNS, Gene Delivery, Gene Promoter, Gene Therapy, LAP, LAT, Latency-Associated Promoter, Latency-Associated Transcript, Light-Sheet Microscopy, Recombinant AAV, Retro-Orbital Injection, Spinal Cord, Viral vector, iDisco+",

author = "Maturana, {Carola J.} and Verpeut, {Jessica L.} and Pisano, {Thomas J.} and Dhanerawala, {Zahra M.} and Andrew Esteves and Enquist, {Lynn W.} and Engel, {Esteban A.}",

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year = "2020",

month = jun,

day = "12",

doi = "10.1016/j.omtm.2020.04.004",

language = "English (US)",

volume = "17",

pages = "843--857",

journal = "Molecular Therapy - Methods and Clinical Development",

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T1 - Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS

AU - Maturana, Carola J.

AU - Verpeut, Jessica L.

AU - Pisano, Thomas J.

AU - Dhanerawala, Zahra M.

AU - Esteves, Andrew

AU - Enquist, Lynn W.

AU - Engel, Esteban A.

PY - 2020/6/12

Y1 - 2020/6/12

N2 - Recombinant adeno-associated viruses (rAAVs) are used as gene therapy vectors. However, they have a limited payload capacity and lack small, long-lasting gene promoters for single-dose gene therapies. We identified small herpesvirus latency-associated promoters showing stable, pan-neuronal transgene transcription and translation in vivo for 6 months after AAV transduction.

AB - Recombinant adeno-associated viruses (rAAVs) are used as gene therapy vectors. However, they have a limited payload capacity and lack small, long-lasting gene promoters for single-dose gene therapies. We identified small herpesvirus latency-associated promoters showing stable, pan-neuronal transgene transcription and translation in vivo for 6 months after AAV transduction.

KW - AAV

KW - Brain

KW - CNS

KW - Gene Delivery

KW - Gene Promoter

KW - Gene Therapy

KW - LAP

KW - LAT

KW - Latency-Associated Promoter

KW - Latency-Associated Transcript

KW - Light-Sheet Microscopy

KW - Recombinant AAV

KW - Retro-Orbital Injection

KW - Spinal Cord

KW - Viral vector

KW - iDisco+

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SN - 2329-0501

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SP - 843

EP - 857

JO - Molecular Therapy - Methods and Clinical Development

JF - Molecular Therapy - Methods and Clinical Development

ER -

Small Alphaherpesvirus Latency-Associated Promoters Drive Efficient and Long-Term Transgene Expression in the CNS

Abstract

Keywords

ASJC Scopus subject areas

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