Therapeutic in vivo genome editing: innovations and challenges in rAAV vector-based CRISPR delivery

No abstract is available for this paper.

• Publication year

  2025

• Venue

  Gene Therapy

• Publication date

  2025-11-12

• Fields of study

  Medicine, Engineering

• Identifiers
  DOI 10.1038/s41434-025-00573-2PMID 41224955PMCID PMC12932096
• External record

  Open on Semantic Scholar

• Source metadata

  Semantic Scholar, PubMed

• No claims are published for this paper.

• No concepts are published for this paper.

• Adeno-Associated Virus Vectors in Retinal Gene Therapy: Challenges, Innovations, and Future Directions

  2025cited by this paper
• Viral and non-viral vectors in gene therapy: current state and clinical perspectives

  2025cited by this paper
• Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease.

  2025cited by this paper
• Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of TOR1A DYT1 allele in a xenograft mouse model

  2025cited by this paper
• Rational engineering of minimally immunogenic nucleases for gene therapy

  2025cited by this paper
• Mismatch prime editing gRNA increased efficiency and reduced indels

  2025cited by this paper
• In vivo applications and toxicities of AAV-based gene therapies in rare diseases

  2025cited by this paper
• SpCas9-HF1 enhances accuracy of cell cycle-dependent genome editing by increasing HDR efficiency, and by reducing off-target effects and indel rates

  2024cited by this paper
• Engineered IscB-ωRNA system with improved base editing efficiency for disease correction via single AAV delivery in mice.

  2024cited by this paper
• Effective genome editing with an enhanced ISDra2 TnpB system and deep learning-predicted ωRNAs

  2024cited by this paper
• Efficient Rescue of Retinal Degeneration in Pde6a Mice by Engineered Base Editing and Prime Editing

  2024cited by this paper
• Engineered IscB–ωRNA system with expanded target range for base editing

  2024cited by this paper
• Adeno-Associated Virus Engineering and Load Strategy for Tropism Modification, Immune Evasion and Enhanced Transgene Expression

  2024cited by this paper
• nCas9 Engineering for Improved Target Interaction Presents an Effective Strategy to Enhance Base Editing

  2024cited by this paper
• Gene Editing for CEP290-Associated Retinal Degeneration.

  2024cited by this paper
• CRISPR base editing-mediated correction of a tau mutation rescues cognitive decline in a mouse model of tauopathy

  2024cited by this paper
• Adeno-associated virus as a delivery vector for gene therapy of human diseases

  2024cited by this paper
• Clinical Pharmacology Perspective on Development of Adeno‐Associated Virus Vector‐Based Retina Gene Therapy

  2024cited by this paper
• Therapeutic In Vivo Gene Editing Achieved by a Hypercompact CRISPR‐Cas12f1 System Delivered with All‐in‐One Adeno‐Associated Virus

  2024cited by this paper
• The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner

  2023cited by this paper
• In vivo treatment of tyrosinaemia with hypercompact Cas12f1

  2023cited by this paper
• Various AAV Serotypes and Their Applications in Gene Therapy: An Overview

  2023cited by this paper
• Efficient prime editing in mouse brain, liver and heart with dual AAVs

  2023cited by this paper
• mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy

  2023cited by this paper
• Vision rescue via unconstrained in vivo prime editing in degenerating neural retinas

  2023influential reference
• Visual function restoration in a mouse model of Leber congenital amaurosis via therapeutic base editing

  2022cited by this paper
• Two high-fidelity variants: efSaCas9 and SaCas9-HF, which one is better?

  2022cited by this paper
• Protein Splicing of Inteins: A Powerful Tool in Synthetic Biology

  2022cited by this paper
• Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors

  2022cited by this paper
• Immune Responses and Immunosuppressive Strategies for Adeno-Associated Virus-Based Gene Therapy for Treatment of Central Nervous System Disorders: Current Knowledge and Approaches

  2022cited by this paper
• Systematic multi-trait AAV capsid engineering for efficient gene delivery

  2022cited by this paper
• Adenine Base Editing In Vivo with a Single Adeno-Associated Virus Vector

  2022influential reference
• Recent advances in CRISPR technologies for genome editing

  2021cited by this paper
• Enhanced specificity mutations perturb allosteric signaling in CRISPR-Cas9

  2021cited by this paper
• Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy

  2021cited by this paper
• Directed evolution of a family of AAV capsid variants enabling potent muscle-directed gene delivery across species.

  2021cited by this paper
• Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

  2021cited by this paper
• Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases

  2021cited by this paper
• Phage-assisted evolution of an adenine base editor with improved Cas domain compatibility and activity

  2020cited by this paper
• CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.

  2020cited by this paper
• Identification of a myotropic AAV by massively parallel in vivo evaluation of barcoded capsid variants

  2020cited by this paper
• Immunity to Cas9 as an Obstacle to Persistent Genome Editing.

  2020cited by this paper
• AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9

  2020cited by this paper
• Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

  2020cited by this paper
• Treatment of a Mouse Model of ALS by In Vivo Base Editing.

  2020cited by this paper
• Engineering the AAV capsid to evade immune responses.

  2019cited by this paper
• Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses

  2019cited by this paper
• Long-Term Effects of In Vivo Genome Editing in the Mouse Retina Using Campylobacter jejuni Cas9 Expressed via Adeno-Associated Virus.

  2019cited by this paper
• Search-and-replace genome editing without double-strand breaks or donor DNA

  2019cited by this paper
• Targeted Transgene Activation in the Brain Tissue by Systemic Delivery of Engineered AAV1 Expressing CRISPRa

  2019cited by this paper
• Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

  2018cited by this paper
• Functional Rescue of Dystrophin Deficiency in Mice Caused by Frameshift Mutations Using Campylobacter jejuni Cas9

  2018cited by this paper
• A communal catalogue reveals Earth’s multiscale microbial diversity

  2017cited by this paper
• Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

  2016cited by this paper
• Development of an intein-mediated split–Cas9 system for gene therapy

  2015cited by this paper
• A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity

  2012cited by this paper

• Integrative Regeneration of Articular Cartilage Using Mesenchymal Stem Cells and Gene Therapy: A Precision Medicine Approach

  2026cites this paper