ReviewMolecular therapy : the journal of the American Society of Gene Therapy2023
Advances in gene therapy hold promise for treating hereditary hearing loss.
Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.
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Who cites it
62 citing papers in PubMed, 112 citations in OpenAlex.
- Efficient Endolymphatic Sac-Directed Gene Delivery Using AAV8BP2 and Posterior Semicircular Canal Injection.International journal of molecular sciences · 2026Article
- The Gut-Ear Axis: From Dysbiosis to Auditory and Vestibular Disorders.International journal of molecular sciences · 2026Review
- Genetic Findings in Seven Cochlear Implanted Patients with Severe-to-Profound Hearing Loss.Genes · 2026Article
- Re-administration of AAV-mediated gene therapy for OTOF-related deafness: a single-arm trial.Nature medicine · 2026Article
- Gene Editing Technologies for Hereditary Hearing Loss: Prospects and Challenges.Neuroscience bulletin · 2026Review
- Polygenic Contribution to Sensorineural Hearing Loss Implicates Novel Risk Loci and Convergence with Congenital Hearing Loss Genes.Journal of the Association for Research in Otolaryngology : JARO · 2026Article
- Long-term restoration of auditory function in a DFNA2 mouse model by adenine base editing.EMBO molecular medicine · 2026Article
- Identification and Functional Characterization of a Novel POU3F4 Frameshift Mutation in a Chinese Family.Life (Basel, Switzerland) · 2026Article
- Capsid-engineered AAV vector overcomes a key intracellular barrier and efficiently transduces spiral ganglion neurons in adult mice.Molecular therapy. Advances · 2026Article
- Bioactive hydrogels for the reconstruction of sensorineural hearing loss.Bioactive materials · 2026Review
- GJB2-Related Hearing Loss: Genotype-Phenotype Correlations, Natural History, and Emerging Therapeutic Strategies.International journal of molecular sciences · 2026Review
- Advancing Gene Therapy for Pancreatitis: From Genetic Insights to Clinical Translation.Research (Washington, D.C.) · 2026Review
- Transcriptomic Profiling Reveals Divergent Immune Responses to AAV1 and AAV-ie in Mice Inner Ear.International journal of medical sciences · 2026Article
- Engineered Inner Ear Drug Delivery Systems for Hearing Loss Treatment.Research (Washington, D.C.) · 2026Review
- Recent preclinical and clinical advances in gene therapy for hereditary hearing loss.Molecules and cells · 2025Review
- Gene therapy restores auditory function and rescues damaged inner hair cells in an aged Vglut3 knockout mouse model.Gene therapy · 2025Article
- Optimized in vivo base editing restores auditory function in a DFNA15 mouse model.Nature communications · 2025Article
- Rapid cochlear gene therapy in adult deaf mice:Molecular therapy. Methods & clinical development · 2025Article
- Delivery of AAV vectors to the superior olivary complex enables efficient adult cochlear transduction via axonal transport.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- geneEX: An Integrated Phenotype-Driven Algorithm for Rapid Identification of Causative Variants in Monogenic Disorders.Molecular genetics & genomic medicine · 2025Article
2 more citing papers are in PubMed but not listed here.
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4 authors at 1 institution in 1 country.
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Abstract
Gene therapy focuses on genetic modification to produce therapeutic effects or treat diseases by repairing or reconstructing genetic material, thus being expected to be the most promising therapeutic strategy for genetic disorders. Due to the growing attention to hearing impairment, an increasing amount of research is attempting to utilize gene therapy for hereditary hearing loss (HHL), an important monogenic disease and the most common type of congenital deafness. Several gene therapy clinical trials for HHL have recently been approved, and, additionally, CRISPR-Cas tools have been attempted for HHL treatment. Therefore, in order to further advance the development of inner ear gene therapy and promote its broad application in other forms of genetic disease, it is imperative to review the progress of gene therapy for HHL. Herein, we address three main gene therapy strategies (gene replacement, gene suppression, and gene editing), summarizing the strategy that is most appropriate for particular monogenic diseases based on different pathogenic mechanisms, and then focusing on their successful applications for HHL in preclinical trials. Finally, we elaborate on the challenges and outlooks of gene therapy for HHL.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.