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Hereditary hearing loss is a genetically heterogeneous condition that affects millions of people worldwide and has limited curative treatment options. Recent advancements in gene therapy have opened promising avenues for correcting the underlying genetic defects in the inner ear. This review summarizes the key developments in vector platforms, delivery strategies, target genes, preclinical models, and clinical trials relevant to both gene supplementation and gene editing approaches, as well as future directions. Adeno-associated virus vectors have emerged as the leading platform for inner ear gene transfer, owing to their safety and efficacy. Clinical programs, such as those targeting OTOF variants, are currently underway and are supported by robust preclinical data. Additionally, genome editing technologies, including CRISPR/Cas9-mediated nonhomologous end joining, base editing, and prime editing, offer variant-specific therapeutic potential. Despite these advances, challenges remain in expanding the therapeutic window, ensuring long-term safety, and establishing ethical and regulatory frameworks for their use.

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Genome-wide statistical evidence elucidates candidate factors of life expectancy in dogs Won Hee Ko, Sangil Kim, Alix Catry, Je-Yoel Cho, Seunggwan Shin p. 1-7
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(The) disordered effector RipAO of Ralstonia solanacearum destabilizes microtubule networks in Nicotiana benthamiana cells Hyelim Jeon, Wanhui Kim, Cécile Segonzac p. 1-14
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Brief guide to immunostaining Gyutae Park, Sieun S. Kim, Jiwon Shim, Seung-Jae V. Lee p. 1-5
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참고문헌 (76건) : 자료제공( 네이버학술정보 )

참고문헌 목록에 대한 테이블로 번호, 참고문헌, 국회도서관 소장유무로 구성되어 있습니다.
번호 참고문헌 국회도서관 소장유무
1 Principles of Local Drug Delivery to the Inner Ear 미소장
2 Self-complementary AAV Vectors; Advances and Applications 미소장
3 HOX-GFP and WOX-GFP lentivirus vectors for inner ear gene transfer 미소장
4 Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness 미소장
5 Novel Mutations in the Connexin 26 Gene (GJB2) That Cause Autosomal Recessive (DFNB1) Hearing Loss 미소장
6 Sound Strategies for Hearing Restoration 미소장
7 Carrier Testing for Severe Childhood Recessive Diseases by Next-Generation Sequencing 미소장
8 Adeno-associated virus-mediated gene transfer to hair cells and support cells of the murine cochlea 미소장
9 Otoferlin, Defective in a Human Deafness Form, Is Essential for Exocytosis at the Auditory Ribbon Synapse 미소장
10 Unmyelinated type II afferent neurons report cochlear damage 미소장
11 Perinatal Gjb2 gene transfer rescues hearing in a mouse model of hereditary deafness 미소장
12 Gene Expression in the Mammalian Cochlea: A Study of Multiple Vector Systems 미소장
13 In Vivo–Directed Evolution of a New Adeno-Associated Virus for Therapeutic Outer Retinal Gene Delivery from the Vitreous 미소장
14 Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain 미소장
15 A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear 미소장
16 CRISPR/Cas9 targeting events cause complex deletions and insertions at 17 sites in the mouse genome 미소장
17 Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems 미소장
18 Intravenous rAAV2/9 injection for murine cochlear gene delivery 미소장
19 Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment 미소장
20 Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents 미소장
21 Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN 미소장
22 Anchor peptide captures, targets, and loads exosomes of diverse origins for diagnostics and therapy 미소장
23 CRISPR–Cas9 genome editing induces a p53-mediated DNA damage response 미소장
24 Enhanced viral-mediated cochlear gene delivery in adult mice by combining canal fenestration with round window membrane inoculation 미소장
25 Repair of double-strand breaks induced by CRISPR–Cas9 leads to large deletions and complex rearrangements 미소장
26 Gene Therapy in Mouse Models of Deafness and Balance Dysfunction 미소장
27 Gene Transfer with AAV9-PHP.B Rescues Hearing in a Mouse Model of Usher Syndrome 3A and Transduces Hair Cells in a Non-human Primate 미소장
28 A dual‐AAV approach restores fast exocytosis and partially rescues auditory function in deaf otoferlin knock‐out mice 미소장
29 Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model 미소장
30 Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality 미소장
31 AAV-ie enables safe and efficient gene transfer to inner ear cells 미소장
32 Screened AAV variants permit efficient transduction access to supporting cells and hair cells 미소장
33 Search-and-replace genome editing without double-strand breaks or donor DNA 미소장
34 Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction 미소장
35 Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae 미소장
36 In vivo base editing restores sensory transduction and transiently improves auditory function in a mouse model of recessive deafness 미소장
37 Direct Delivery of Antisense Oligonucleotides to the Middle and Inner Ear Improves Hearing and Balance in Usher Mice 미소장
38 Gene therapy via canalostomy approach preserves auditory and vestibular functions in a mouse model of Jervell and Lange-Nielsen syndrome type 2 미소장
39 Refining surgical techniques for efficient posterior semicircular canal gene delivery in the adult mammalian inner ear with minimal hearing loss 미소장
40 Differential genetic diagnoses of adult post-lingual hearing loss according to the audiogram pattern and novel candidate gene evaluation 미소장
41 COCH-related autosomal dominant nonsyndromic hearing loss: a phenotype–genotype study 미소장
42 Dual-vector gene therapy restores cochlear amplification and auditory sensitivity in a mouse model of DFNB16 hearing loss 미소장
43 In vivo outer hair cell gene editing ameliorates progressive hearing loss in dominant-negative Kcnq4 murine model 미소장
44 Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice 미소장
45 Hearing of Otof-deficient mice restored by trans-splicing of N- and C-terminal otoferlin 미소장
46 Advances in gene therapy hold promise for treating hereditary hearing loss 미소장
47 Efficient prime editing in mouse brain, liver and heart with dual AAVs 미소장
48 Deafness: from genetic architecture to gene therapy 미소장
49 Rescue of auditory function by a single administration of AAV-TMPRSS3 gene therapy in aged mice of human recessive deafness DFNB8 미소장
50 Selection of viral capsids and promoters affects the efficacy of rescue of Tmprss3-deficient cochlea 미소장
51 Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo 미소장
52 Phage-assisted evolution and protein engineering yield compact, efficient prime editors 미소장
53 Preclinical evaluation of the efficacy and safety of AAV1-hOTOF in mice and nonhuman primates 미소장
54 Preclinical Efficacy And Safety Evaluation of AAV‐OTOF in DFNB9 Mouse Model And Nonhuman Primate 미소장
55 Distributional comparison of different AAV vectors after unilateral cochlear administration 미소장
56 AAV‐Mediated Gene Therapy Restores Hearing in Patients with DFNB9 Deafness 미소장
57 Clarin-2 gene supplementation durably preserves hearing in a model of progressive hearing loss 미소장
58 AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial 미소장
59 AAV‐mediated Gene Cocktails Enhance Supporting Cell Reprogramming and Hair Cell Regeneration 미소장
60 Bilateral gene therapy in children with autosomal recessive deafness 9: single-arm trial results 미소장
61 Targeted genome editing restores auditory function in adult mice with progressive hearing loss caused by a human microRNA mutation 미소장
62 A base editor for the long-term restoration of auditory function in mice with recessive profound deafness 미소장
63 Reprogramming with Atoh1, Gfi1, and Pou4f3 promotes hair cell regeneration in the adult organ of Corti 미소장
64 PCDH15 Dual-AAV Gene Therapy for Deafness and Blindness in Usher Syndrome Type 1F Models 미소장
65 Common genetic etiologies of sensorineural hearing loss in Koreans 미소장
66 Cell-specific delivery of GJB2 restores auditory function in mouse models of DFNB1 deafness and mediates appropriate expression in NHP cochlea 미소장
67 Bi-allelic variants of SEMA3F are associated with non-syndromic hearing loss 미소장
68 Combined AAV-mediated specific Gjb2 expression restores hearing in DFNB1 mouse models 미소장
69 Systematic genetic assessment of hearing loss using whole-genome sequencing identifies pathogenic variants 미소장
70 Bystander editing caused by adenine base editors interferes with visual function restoration in a mouse model of Leber congenital amaurosis 미소장
71 Deciphering enhancers of hearing loss genes for efficient and targeted gene therapy of hereditary deafness 미소장
72 A suite of enhancer AAVs and transgenic mouse lines for genetic access to cortical cell types 미소장
73 AAV gene therapy for autosomal recessive deafness 9: a single-arm trial 미소장
74 Identification and application of cell-type-specific enhancers for the macaque brain 미소장
75 AAV gene therapy rescues hearing and balance in a model of CLIC5 deafness 미소장
76 (Reference title not available) 미소장

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