The Rise of Cochlear Gene Therapy DOI Creative Commons
Lukas D. Landegger, Ellen Reisinger, François Lallemend

и другие.

Molecular Therapy, Год журнала: 2024, Номер unknown

Опубликована: Ноя. 1, 2024

Recent evidence provides strong support for the safe and effective use of gene therapy in humans with hearing loss. By means a single local injection set adeno-associated virus (AAV) vectors, was partially restored several children neurosensory nonsyndromic autosomal recessive deafness 9 (DFNB9), harboring variants OTOF gene. Current research focuses on refining endoscopic transmastoid procedures to reduce risks side effects, as emerging suggests bidirectional fluid exchanges between ear brain. Moreover, editing approaches novel AAV capsids are successfully tested animal models will likely lead enhanced targeting cochlea. Here, we cover recent advances cochlear therapy, provide an overview translational potential these new existing future clinical trials, highlight implications that remain be determined their application humans.Graphical abstract

Язык: Английский

Pharmacological Approaches to Hearing Loss DOI Creative Commons
Christopher R. Cederroth, Jonas Dyhrfjeld‐Johnsen, Barbara Canlon

и другие.

Pharmacological Reviews, Год журнала: 2024, Номер 76(6), С. 1063 - 1088

Опубликована: Окт. 16, 2024

Hearing disorders pose significant challenges to individuals experiencing them and their overall quality of life, emphasizing the critical need for advanced pharmacological approaches address these conditions. Current treatment options often focus on amplification devices, cochlear implants, or other rehabilitative therapies, leaving a substantial gap regarding effective interventions. Advancements in our understanding molecular cellular mechanisms involved hearing induced by noise, aging, ototoxicity have opened new avenues drug development, some which led numerous clinical trials, with promising results. The development optimal delivery solutions animals humans can also enhance targeted medications ear. Moreover, large genome studies contributing genetic loss combined technologies animal shown great potential increase etiologies loss. auditory system exhibits circadian rhythms temporal variations its physiology, vulnerability insults, responsiveness treatments. clock are under control glucocorticoid system, preclinical evidence suggests that risk/benefit profile disorder treatments using chronopharmacological would be beneficial. If translatable bedside, such may improve outcome trials. Ongoing research into basis disorders, coupled advancements formulation as well optimized timing administration, holds promise more SIGNIFICANCE STATEMENT: procedures

Язык: Английский

Процитировано

3

Cellular diversity of human inner ear organoids revealed by single-cell transcriptomics DOI
Mireia Rumbo, Berta Alsina

Development, Год журнала: 2024, Номер 151(23)

Опубликована: Ноя. 29, 2024

ABSTRACT Human inner ear organoids are three-dimensional tissular structures grown in vitro that recapitulate some aspects of the fetal and allow differentiation cell types. These offer a system which to study human development, mutations causing hearing loss vertigo, new therapeutic drugs. However, extent such mimic vivo development cellular composition remains unclear. Several recent studies have performed single-cell transcriptomics on interrogate heterogeneity, reveal developmental trajectories sensory lineages compare organoid-derived vesicles developing ear. Here, we discuss insights provided by these analyses help define paths investigation understand development.

Язык: Английский

Процитировано

1

Synthetically programming natural cell-cell communication pathways for tissue engineering DOI

Leah A. Wallach,

Connor D. Thomas,

Pulin Li

и другие.

Current Opinion in Biomedical Engineering, Год журнала: 2024, Номер 32, С. 100554 - 100554

Опубликована: Авг. 24, 2024

Язык: Английский

Процитировано

0

The Rise of Cochlear Gene Therapy DOI Creative Commons
Lukas D. Landegger, Ellen Reisinger, François Lallemend

и другие.

Molecular Therapy, Год журнала: 2024, Номер unknown

Опубликована: Ноя. 1, 2024

Recent evidence provides strong support for the safe and effective use of gene therapy in humans with hearing loss. By means a single local injection set adeno-associated virus (AAV) vectors, was partially restored several children neurosensory nonsyndromic autosomal recessive deafness 9 (DFNB9), harboring variants OTOF gene. Current research focuses on refining endoscopic transmastoid procedures to reduce risks side effects, as emerging suggests bidirectional fluid exchanges between ear brain. Moreover, editing approaches novel AAV capsids are successfully tested animal models will likely lead enhanced targeting cochlea. Here, we cover recent advances cochlear therapy, provide an overview translational potential these new existing future clinical trials, highlight implications that remain be determined their application humans.Graphical abstract

Язык: Английский

Процитировано

0