Abstract
The application of gene therapy to the inner ear is an emerging field of study. Most studies report the expression of marker genes (e.g., galactosidase) within the tissues of the cochlea. The first biologic response of an inner ear tissue (i.e., auditory neurons) to transduction by a gene therapy vector expressing a therapeutic gene (a herpes amplicon vector containing a BDNF gene) was observed in spiral explants obtained from early postnatal rat cochleae. This study was important because it demonstrated the feasibility of a gene augmentation approach to treat traumatized cochleae. Long-term expression of transduced or transfected genes in cochlear tissues have been obtained with adenovirus, adeno-associated virus, and herpes amplicon vectors. The herpes amplicon vector (i.e., HSVbdnflac) that evoked a biologic response in vitro has also been successfully used to support the survival of auditory neurons in vivo following loss of the auditory hair cells (i.e., loss of trophic factor). Gene therapy has been successfully applied to the cochlea of a laboratory animal, and future studies will define the types of vectors and therapeutic genes that will work best for the treatment of inner ear diseases in the clinic.
| Original language | English |
|---|---|
| Pages (from-to) | 345-360 |
| Number of pages | 16 |
| Journal | Annals of the New York Academy of Sciences |
| Volume | 884 |
| State | Published - Jan 1 1999 |
| Externally published | Yes |
Fingerprint
ASJC Scopus subject areas
- Biochemistry, Genetics and Molecular Biology(all)
Cite this
Gene therapy in the inner ear : Mechanisms and clinical implications. / Van De Water, Thomas R; Staecker, Hinrich; Halterman, Marc W.; Federoff, Howard J.
In: Annals of the New York Academy of Sciences, Vol. 884, 01.01.1999, p. 345-360.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Gene therapy in the inner ear
T2 - Mechanisms and clinical implications
AU - Van De Water, Thomas R
AU - Staecker, Hinrich
AU - Halterman, Marc W.
AU - Federoff, Howard J.
PY - 1999/1/1
Y1 - 1999/1/1
N2 - The application of gene therapy to the inner ear is an emerging field of study. Most studies report the expression of marker genes (e.g., galactosidase) within the tissues of the cochlea. The first biologic response of an inner ear tissue (i.e., auditory neurons) to transduction by a gene therapy vector expressing a therapeutic gene (a herpes amplicon vector containing a BDNF gene) was observed in spiral explants obtained from early postnatal rat cochleae. This study was important because it demonstrated the feasibility of a gene augmentation approach to treat traumatized cochleae. Long-term expression of transduced or transfected genes in cochlear tissues have been obtained with adenovirus, adeno-associated virus, and herpes amplicon vectors. The herpes amplicon vector (i.e., HSVbdnflac) that evoked a biologic response in vitro has also been successfully used to support the survival of auditory neurons in vivo following loss of the auditory hair cells (i.e., loss of trophic factor). Gene therapy has been successfully applied to the cochlea of a laboratory animal, and future studies will define the types of vectors and therapeutic genes that will work best for the treatment of inner ear diseases in the clinic.
AB - The application of gene therapy to the inner ear is an emerging field of study. Most studies report the expression of marker genes (e.g., galactosidase) within the tissues of the cochlea. The first biologic response of an inner ear tissue (i.e., auditory neurons) to transduction by a gene therapy vector expressing a therapeutic gene (a herpes amplicon vector containing a BDNF gene) was observed in spiral explants obtained from early postnatal rat cochleae. This study was important because it demonstrated the feasibility of a gene augmentation approach to treat traumatized cochleae. Long-term expression of transduced or transfected genes in cochlear tissues have been obtained with adenovirus, adeno-associated virus, and herpes amplicon vectors. The herpes amplicon vector (i.e., HSVbdnflac) that evoked a biologic response in vitro has also been successfully used to support the survival of auditory neurons in vivo following loss of the auditory hair cells (i.e., loss of trophic factor). Gene therapy has been successfully applied to the cochlea of a laboratory animal, and future studies will define the types of vectors and therapeutic genes that will work best for the treatment of inner ear diseases in the clinic.
UR - http://www.scopus.com/inward/record.url?scp=0032787488&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=0032787488&partnerID=8YFLogxK
M3 - Article
C2 - 10842605
AN - SCOPUS:0032787488
VL - 884
SP - 345
EP - 360
JO - Annals of the New York Academy of Sciences
JF - Annals of the New York Academy of Sciences
SN - 0077-8923
ER -