Gene therapy is transforming hereditary hematological disorders. This review summarizes approved gene addition, editing, and silencing strategies for sickle cell disease, thalassemia, and hemophilia, highlights curative potential, and discusses remaining challenges such as immune responses, cost, and accessibility

The authors develop AminiCRoff, a compact dAsCas12f1-based epigenetic silencer compatible with single-AAV delivery. AminiCRoff mediates durable, heritable gene silencing comparable to the larger CRISPRoff and represses endogenous MYC to inhibit tumor cell proliferation, providing a versatile platform for in vivo epigenome editing.

Blancafort and colleagues describe a non-viral polymeric system for the delivery of dCas9-KRAB silencers as ribonucleoprotein (RNP) payloads for EWSR1-FLI1 repression. They demonstrate highly efficient RNP delivery and robust silencing of EWSR1-FLI1 in both cell line and patient-derived xenografts of Ewing sarcoma, accompanied by potent anti-tumor effects.

Structure-guided design combined with protein language model-guided engineering and sgRNA optimization enables the development of a compact and highly efficient CRISPR–Cas12f genome editor. This integrated strategy substantially improves genome-editing activity while preserving high specificity, expanding the therapeutic potential of compact CRISPR systems.

Ouabain triggers cochlear spiral ganglion neuron (SGN) ferroptosis and hearing loss via SCD1 downregulation. MITF directly activates Scd1 transcription, and the MITF–SCD1 axis mitigates SGN ferroptosis and hearing impairment in ototoxic ouabain and cisplatin models, revealing a lipid metabolic vulnerability and therapeutic target for sensorineural hearing loss.

(Molecular Therapy 31, 344–361; February 2023)

Cancer cachexia is a devastating metabolic syndrome for which there are no approved treatments. Li and colleagues developed an RNA-targeted therapy, which ameliorates cachectic symptoms, reduces inflammation, and extends survival in mouse cancer models. The study provides an approach for treating cancer cachexia and paves the road for clinical studies.

LMNA-related congenital muscular dystrophy (L-CMD) is a devastating early-onset muscle disease caused by dysfunctional nuclear lamina. Current models fail to capture the complexity of human muscle pathology, limiting translational progress. This study overcomes this limitation by establishing a robust, human iPSC-based platform for modelling L-CMD and testing gene editing strategies.

A vasoactive intestinal peptide (VIP)-based protein carrier self-assembles with respiratory syncytial virus circular RNA vaccines for intranasal delivery, inducing systemic antibodies, mucosal IgA, and Th1-biased protection in mice. This platform offers a protein-guided strategy for respiratory mucosal RNA vaccination and broadens the application of VIP in vaccine delivery.

Red blood cell-derived extracellular vesicles (RBCEVs) provide a scalable, non-viral platform for gene therapy. High-grade RBCEVs purified using tangential flow filtration can deliver sizable plasmids and mediate sustained in vivo expression of therapeutic proteins, including factor IX and Herceptin, highlighting their potential use for safe and efficient gene therapy.

Liu and colleagues developed a robust in cellulo plasmid DNA replication system in human cells for replicating plasmid-borne adeno-associated virus (AAV) Rep/Cap genes during recombinant AAV (rAAV) production. This new approach not only enables a 10- to 20-fold plasmid reduction to significantly lower manufacturing costs but also substantially enhances rAAV potency, titer, and purity.

MNK1 acts as a structural shield on MYH9, preventing YAP1-mediated platelet activation. Developing MD2 to lock this MNK1-MYH9 complex introduces a safe antithrombotic strategy, shifting the therapeutic paradigm from kinase inhibition to stabilizing protein-protein interactions against immunothrombosis.

Latent HIV reservoirs evade both antiviral therapy and immune surveillance. Luo and colleagues develop a multifunctional fusion protein that couples reservoir reactivation with targeted immune engagement and clearance, offering a coordinated strategy to expose and eliminate persistent HIV-infected cells.

This study identifies AAV2.7m8-Myo15 as a safe and efficient vector for transducing cochlear outer hair cells in adult mice via posterior semicircular canal injection. These insights overcome age-related transduction barriers and suggest potential gene therapy strategies for sensorineural hearing loss.

CAR T cells engineered to secrete tumor-localized anti-EpCAM bispecific T cell engagers (BTCEs) overcome antigen escape and heterogeneity across multiple epithelial carcinomas in preclinical models, achieving complete tumor eradication where conventional single-target CAR T cell therapies often failed, supporting broad translational potential for solid tumor immunotherapy.

Jin and colleagues review recent advances in mRNA vaccines for infectious diseases and cancer, covering sequence optimization, circRNA/saRNA platforms, delivery systems, and clinical translation challenges, while highlighting the role of computational technologies.

Nature Biomedical Engineering, Published online: 07 September 2026; doi:10.1038/s41551-026-01794-5Author Correction: A base editor for the long-term restoration of auditory function in mice with recessive profound deafness

Nature Biomedical Engineering, Published online: 31 August 2026; doi:10.1038/s41551-026-01782-9A protein engineering strategy enables nitric oxide-triggered reactivation of proteins using genetically encoded caged amino acids, allowing inflammation-localized control of protein activity, viral gene delivery and biosensing in vivo.

Nature Nanotechnology, Published online: 08 September 2026; doi:10.1038/s41565-026-02288-wPublisher Correction: Edge-sharing RuO2 single layer for stable and low overpotential acidic water electrolysis

Nature Nanotechnology, Published online: 08 September 2026; doi:10.1038/s41565-026-02275-1Structured light–matter interaction at the single-photon level is demonstrated in a coupled quantum-dot–micropillar system, enabling cavity-enhanced single-photon emission with spin-locked orbital angular momentum and tunable spin–orbit entanglement.