Gene Therapy最新文献

AAV-mediated delivery of leptin but not adiponectin improves metabolic health in a pre-clinical mouse model of congenital generalised lipodystrophy. aav介导的瘦素而非脂联素的递送改善了先天性全身性脂肪营养不良小鼠临床前模型的代谢健康。

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-10-23 DOI: 10.1038/s41434-026-00646-w

Nadine Sommer, Ahlima Roumane, Mansi Tiwari, Weiping Han, Lora K Heisler, George D Mcilroy, Justin J Rochford

{"title":"AAV-mediated delivery of leptin but not adiponectin improves metabolic health in a pre-clinical mouse model of congenital generalised lipodystrophy.","authors":"Nadine Sommer, Ahlima Roumane, Mansi Tiwari, Weiping Han, Lora K Heisler, George D Mcilroy, Justin J Rochford","doi":"10.1038/s41434-026-00646-w","DOIUrl":"https://doi.org/10.1038/s41434-026-00646-w","url":null,"abstract":"Lipodystrophies are a group of disorders featuring reduced adipose tissue mass or function, which often leads to significant metabolic disease, reduced lifespan and impaired quality of life. Individuals with congenital generalised lipodystrophy (CGL) have severely reduced adipose tissue mass. The loss of healthy systemic lipid storage typically causes hepatic steatosis and lipoatrophic diabetes. In addition, adipocyte-secreted hormones including leptin and adiponectin are dramatically reduced. Leptin has critical roles regulating appetite and broader effects on lipid and glucose metabolism. Daily injection with recombinant leptin is currently the only specific, approved treatment for CGL. The consequences of adiponectin loss in these patients are not fully understood. Likewise, the potential therapeutic benefit of adiponectin delivery is unclear. Here we examine the effect of delivering leptin or adiponectin by adeno-associated virus (AAV) as a potential gene therapy treatment for metabolic disease in CGL using a well-characterised murine model of the condition. AAV-mediated leptin delivery significantly improved hepatic steatosis and hyperinsulinemia. However, adiponectin delivery did not lead to any observed beneficial effects. This demonstrates that gene therapy approaches may have the potential to offer long-term delivery of leptin in individuals with lipodystrophy, without the need for continuous supply of perishable therapeutics and painful daily injections.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-10-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149244070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Prevalence of adeno-associated virus type 3B neutralizing antibodies in cohorts of healthy donors and Wilson's disease adults. 腺相关病毒3B型中和抗体在健康供体和成年威尔逊氏病人群中的流行

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-10-20 DOI: 10.1038/s41434-026-00651-z

Céline Bouquet, Blanche Tamarit, Gloria Gonzalez Aseguinolaza, Veronica Ferrer, Sonia Valero, Anne Douar, Annelise Brossel, Rainard Fuhr, Jean-Philippe Combal

{"title":"Prevalence of adeno-associated virus type 3B neutralizing antibodies in cohorts of healthy donors and Wilson's disease adults.","authors":"Céline Bouquet, Blanche Tamarit, Gloria Gonzalez Aseguinolaza, Veronica Ferrer, Sonia Valero, Anne Douar, Annelise Brossel, Rainard Fuhr, Jean-Philippe Combal","doi":"10.1038/s41434-026-00651-z","DOIUrl":"https://doi.org/10.1038/s41434-026-00651-z","url":null,"abstract":"Adeno-associated virus (AAV) is widely used for liver-directed gene therapy, with AAV serotype 3B (AAV3B) showing particularly high tropism for human hepatocytes. However, pre-existing neutralizing antibodies (NAbs) against AAV represent a major barrier to efficient gene transfer and patient eligibility. We have recently demonstrated that subjects with intermediate NAb levels (1:5-1:45), that would be excluded from clinical trials, can be converted in eligible patients by the administration of IgG-cleaving enzymes. It is thus crucial to assess not only the seroprevalence but also titer distribution. Here, we analyzed anti-AAV3B NAb titers, using a validated cell-based transduction inhibition assay, in healthy adults and patients with Wilson's disease, a hepatic metabolic disorder amenable to AAV3B-mediated gene therapy. Two independent cohorts of healthy adults (n = 200 and n = 580) were analyzed; 33-36% of individuals were NAb positive, and prevalence increased with age. Differences were observed across ethnicities. Importantly, a substantial proportion of seropositive individuals (19%) exhibited intermediate NAb titers. In contrast, only 17.4% of Wilson's disease patients (n = 23) were positive, all with high NAb titers. These findings provide insights into target population eligibility, and titer distribution data support the potential of NAb-reduction approaches to expand access to systemic AAV3B-based gene therapies.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149227672","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Chronic lung infection does not impair lentiviral vector gene transfer in a cystic fibrosis rat model. 慢性肺部感染不影响囊性纤维化大鼠模型中的慢病毒载体基因转移。

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-10-19 DOI: 10.1038/s41434-026-00652-y

Alexandra McCarron, Nicole Reyne, Bernadette Boog, Patricia Cmielewski, Martin Donnelley

{"title":"Chronic lung infection does not impair lentiviral vector gene transfer in a cystic fibrosis rat model.","authors":"Alexandra McCarron, Nicole Reyne, Bernadette Boog, Patricia Cmielewski, Martin Donnelley","doi":"10.1038/s41434-026-00652-y","DOIUrl":"https://doi.org/10.1038/s41434-026-00652-y","url":null,"abstract":"Genetic therapies offer a promising strategy for treating cystic fibrosis (CF) lung disease, particularly for individuals with modulator-ineligible genotypes. However, clinical translation has been slower than anticipated. While CF gene therapy candidates typically demonstrate high efficacy in preclinical studies, all products trialled to date have failed to progress beyond Phase I/II, raising questions about the predictive capacity of current preclinical models. One contributing factor may be the absence of severe muco-obstructive lung disease in commonly used CF rodent models, highlighting the need to evaluate gene vectors under conditions that more closely recapitulate the CF lung environment. In this study, we evaluated a vesicular stomatitis virus glycoprotein G (VSV-G) pseudotyped lentiviral (LV) vector carrying the luciferase reporter gene under conditions of Pseudomonas aeruginosa lung infection in wild-type and CF rats. Bioluminescence imaging was conducted on days 7 and 14 post-LV vector administration to assess lung transgene expression under infected and non-infected conditions. At day 7, there was no significant difference in bioluminescence between P. aeruginosa-infected and non-infected animals (p = 0.537). From day 7 to day 14, bioluminescence decreased 136-fold in non-infected animals (p < 0.0001), compared with a 7-fold decrease in infected animals (p = 0.043), with a significant infection × time interaction indicating that the magnitude of decline was greater in non-infected controls (p = 0.024). These findings demonstrate that lung disease state can modulate transgene expression and persistence, highlighting the importance of evaluating gene therapy vectors under conditions that more closely mimic the CF lung environment.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-10-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149223983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Statistical correlation between strength and mini-dystrophin expression in skeletal muscle after an rAAV9-mini-dystrophin gene therapy candidate in DMD^mdx rats. DMDmdx大鼠rAAV9-mini-dystrophin候选基因治疗后骨骼肌强度与mini-dystrophin表达的统计学相关性

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-10-17 DOI: 10.1038/s41434-026-00648-8

J M Walsh, A McIntosh, J Owens, C Le Guiner, A Lafoux, C Huchet, M Binks, S Neelakantan, V Farrokhi, H Neubert

{"title":"Statistical correlation between strength and mini-dystrophin expression in skeletal muscle after an rAAV9-mini-dystrophin gene therapy candidate in DMDmdx rats.","authors":"J M Walsh, A McIntosh, J Owens, C Le Guiner, A Lafoux, C Huchet, M Binks, S Neelakantan, V Farrokhi, H Neubert","doi":"10.1038/s41434-026-00648-8","DOIUrl":"https://doi.org/10.1038/s41434-026-00648-8","url":null,"abstract":"Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder resulting from loss-of-function mutations in the dystrophin gene, leading to progressive muscle wasting and premature death. We present the mini-dystrophin protein expression-function relationship from a post-hoc analysis of mini-dystrophin quantification and function (strength) data at 3 and 6 months in biceps femoris and pectoralis muscles from a DMD rat model (DMDmdx) treated with fordadistrogene movaparvovec, an adeno-associated virus serotype 9 gene therapy containing miniaturised dystrophin. Four treatment groups of ~7-9-week-old DMDmdx rats and two control groups (one wild-type and one DMDmdx group) of ~7-9-week-old DMDmdx rats received fordadistrogene movaparvovec (1E + 13, 3E + 13, 1E + 14 or 3E + 14 vg/kg) or formulation buffer, respectively. Muscle samples were harvested at 3 or 6 months post-administration. Previously reported treatment of DMDmdx rats with fordadistrogene movaparvovec increased body weight, improved clinical chemistry parameters, ameliorated skeletal and cardiac muscle histopathology and improved muscle and cardiac function, all in a dose-dependent manner. Data from this post-hoc analysis indicate that expression of the mini-dystrophin protein levels is sustained or increased (at lower doses) from 3 to 6 months post-dose in DMDmdx rats treated with fordadistrogene movaparvovec. Taken together, this suggests a correlation between mini-dystrophin protein expression and function in the DMDmdx rat model 3 and 6 months after fordadistrogene movaparvovec.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149216196","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Correction: CRISPR/Cas9-mediated gene correction of Wilson disease H1069Q point mutation in patient-specific induced pluripotent stem cells. 纠正：CRISPR/ cas9介导的威尔森病H1069Q点突变在患者特异性诱导多能干细胞中的基因纠正。

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-10-02 DOI: 10.1038/s41434-026-00647-9

Viktoria Iwan, Oksana Nadzemova, Matthias Weiand, Andree Zibert, Hartmut H Schmidt, Phil-Robin Tepasse, Robert Schierwagen, Jonel Trebicka, Vanessa Sandfort

引用次数: 0

Local targeted suppression of neocortical spreading depolarization amplitude by viral expression of KCa3.1 channels. 病毒表达KCa3.1通道局部靶向抑制新皮层扩张性去极化振幅

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-09-29 DOI: 10.1038/s41434-026-00645-x

Ilya A Oblasov, Maria P Smirnova, Anastasia A Borodinova, Alena B Zuzina, Ivan A Smirnov, Pavel M Balaban, Lyudmila V Vinogradova, Evgeny S Nikitin

{"title":"Local targeted suppression of neocortical spreading depolarization amplitude by viral expression of KCa3.1 channels.","authors":"Ilya A Oblasov, Maria P Smirnova, Anastasia A Borodinova, Alena B Zuzina, Ivan A Smirnov, Pavel M Balaban, Lyudmila V Vinogradova, Evgeny S Nikitin","doi":"10.1038/s41434-026-00645-x","DOIUrl":"https://doi.org/10.1038/s41434-026-00645-x","url":null,"abstract":"Cortical spreading depolarization (SD) underlies migraine, frequently co-occurs with seizures, and is associated with poor prognosis after stroke. In rodent models, most genetic therapies for epilepsy suppress seizures by enhancing neuronal K⁺ conductance. Although seizures and SD often co-occur and SD causes temporary neuronal silencing, it remains unknown whether K⁺-conductance-enhancing gene therapy affects SD. Here, we present a genetic method for targeted downregulation of SD using adeno-associated virus to express KCNN4, which encodes the Ca²⁺-gated KCa3.1 channels. We tested the effect of KCNN4 expression in rodent neocortex using patch-clamp electrophysiology, functional imaging in brain slices, and field potential recording in vivo. KCNN4 expression reduced acute seizure-like activity, lowered SD amplitude several-fold both in vivo and in vitro, and slowed SD propagation in brain slices, where epileptiform activity paradoxically reappeared faster after SD. Moreover, SD triggered a Ca²⁺ rebound upon restoration of epileptiform activity, which was counterbalanced by KCa3.1-mediated afterhyperpolarization. Targeted suppression of SD amplitude using KCNN4 expression provides a proof-of-principle for potential gene therapy models for disorders complicated by SD. Reducing SD via increased neuronal K⁺ conductance may have a side effect worth considering in gene therapy for epilepsy, as some literature suggest that SD itself may terminate seizures.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149080679","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Rational selection of SpCas9 and variant-derived adenine base editors for human genome editing. SpCas9和变异衍生腺嘌呤碱基编辑器的合理选择

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-09-27 DOI: 10.1038/s41434-026-00639-9

Jiahua Wang, Leben He, Lin Li, Yujing Zhang, Yuanyuan Wang, Yuejun Yang, Zongming Song, Yuanbo Liang, Jin Yang, Feng Gu

{"title":"Rational selection of SpCas9 and variant-derived adenine base editors for human genome editing.","authors":"Jiahua Wang, Leben He, Lin Li, Yujing Zhang, Yuanyuan Wang, Yuejun Yang, Zongming Song, Yuanbo Liang, Jin Yang, Feng Gu","doi":"10.1038/s41434-026-00639-9","DOIUrl":"https://doi.org/10.1038/s41434-026-00639-9","url":null,"abstract":"Adenine base editors (ABEs), composed of Cas9 (i.e., SpCas9) nickase, adenosine deaminase, and sgRNA, can convert A•T base pairs to G•C base pairs in the genome without causing DNA double-strand breaks, thereby offering broad application prospects. The strict protospacer adjacent motif requirement and off-target effects of wild-type SpCas9 notably restricted its application until the identification of two SpCas9 variants, SpCas9-NG and SpRY, which have overcome these limitations. However, how to select appropriate ABEs from various SpCas9 variants remains unclear. In this study, we evaluated NGG-ABE8e, NG-ABE8e, and SpRY-ABE8e in human cells and explored the impact of sgRNA design on their performance. The results indicated that NGG-ABE8e showed higher editing activity at most tested NGG-PAM sites, whereas SpRY-ABE8e showed stronger mismatch discrimination and lower detectable A-to-G editing at the selected predicted off-target sites with NGG-PAM sites. Regarding sgRNA design, we found that spacer length and 5' guanine (G) addition affected editing outcomes in a site- and editor-dependent manner. Notably, adding an extra 5' G was not uniformly beneficial and could reduce editing efficiency, especially when the native spacer already began with G. In conclusion, this study provides insights into selecting optimal ABEs and designing effective sgRNAs for the treatment of human genetic diseases.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-09-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149059729","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies. 患者、消费者和社会价值观、对高成本基因疗法的看法和偏好。

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-09-17 DOI: 10.1038/s41434-026-00643-z

Amanda Rush, Maria Gomez, Kristine Pierce, De Abreu Lourenco Richard, Kylie Mason, Kirsten Howard, Garry Lynch, Pippy Walker, Rosalie Viney, Jo Watson

{"title":"Patient, consumer and societal values, perceptions and preferences on high-cost gene therapies.","authors":"Amanda Rush, Maria Gomez, Kristine Pierce, De Abreu Lourenco Richard, Kylie Mason, Kirsten Howard, Garry Lynch, Pippy Walker, Rosalie Viney, Jo Watson","doi":"10.1038/s41434-026-00643-z","DOIUrl":"https://doi.org/10.1038/s41434-026-00643-z","url":null,"abstract":"Novel cell and gene therapies hold great potential to alter the clinical course of a range of chronic diseases, yet uncertainties remain regarding their long-term efficacy and impact. These uncertainties pose challenges for Health Technology Assessment (HTA) committees, which systematically assess health interventions seeking public subsidy. Haematological conditions are a common target for gene therapies; however, the perspectives of those both directly and indirectly impacted by funding decisions for therapies in this disease area have not been investigated. To support future HTA considerations of gene therapies, we explored the perspectives of individuals living with haematological conditions who may become eligible for such treatments, alongside their caregivers and members of the general population in Australia. We undertook interviews and focus groups with 55 participants with lived experience and 32 general population members to elicit responses on what might influence decision making on gene therapies; the potential benefits or harms of gene therapies; any perceived barriers to gene therapy access; and/or uncertainties associated with the respective gene therapy. Here, we describe the parallel perspectives of health consumers and the general population, providing five key areas of consideration for HTA decision-making: individual patient and disease factors; geographical, financial and clinical access; balancing disease and quality of life benefits with adverse effects; individual and health system costs; and remaining treatment uncertainties. These will require resolution before widespread acceptability and uptake of gene therapy.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-09-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148989754","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Intratumor delivery of a novel plasmid combination induces high levels of metastatic melanoma regression. 肿瘤内递送一种新的质粒组合可诱导高水平的转移性黑色素瘤消退。

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-09-16 DOI: 10.1038/s41434-026-00644-y

Loree C Heller, Julie S Singh, Jody C Synowiec, Mark J Jaroszeski, Alex Otten, Richard Heller

引用次数: 0

Culture media alter retinal organoid physiology promoting AAV transduction and retinal ganglion cell survival. 培养基改变视网膜类器官生理，促进AAV转导和视网膜神经节细胞存活。

IF 5.9 3区医学

Gene Therapy Pub Date : 2026-09-05 DOI: 10.1038/s41434-026-00642-0

Michelle O'Hara-Wright, Benjamin Y Lim, Melissa M Mangala, Vivienne Kaiser, Emilie Wong, Deborah Aubin, Victoria Nemeruck, Hallur Reynisson, Fatemeh Doroudian, Otilia P Y Chan, Nader Aryamanesh, Joao A Paulo, Stefano Palomba, Mehdi Mirzaei, Samantha L Ginn, Anai Gonzalez-Cordero

{"title":"Culture media alter retinal organoid physiology promoting AAV transduction and retinal ganglion cell survival.","authors":"Michelle O'Hara-Wright, Benjamin Y Lim, Melissa M Mangala, Vivienne Kaiser, Emilie Wong, Deborah Aubin, Victoria Nemeruck, Hallur Reynisson, Fatemeh Doroudian, Otilia P Y Chan, Nader Aryamanesh, Joao A Paulo, Stefano Palomba, Mehdi Mirzaei, Samantha L Ginn, Anai Gonzalez-Cordero","doi":"10.1038/s41434-026-00642-0","DOIUrl":"https://doi.org/10.1038/s41434-026-00642-0","url":null,"abstract":"Human iPSC-derived retinal organoids offer a human-relevant platform for inherited retinal disease (IRD) gene therapy, yet robust AAV transduction in vitro remains challenging. Here we show that culture in BrainPhys™ (BP) medium markedly enhances AAV-mediated gene delivery. Brief BP exposure during the transduction window improves uptake, whereas continuous BP culture yields the strongest effects, indicating contributions from acute entry processes and longer-term cellular remodelling. Quantitative proteomics reveal coordinated upregulation of viral entry and trafficking mediators, including integrins that form αVβ5, and the trans-Golgi entry factor GPR108, together with enrichment of endosomal/ER-Golgi transport proteins, providing a mechanistic basis for enhanced vector processing. BP concurrently promotes neuronal maturation and metabolism, with pronounced increases in neurotrophic and synaptic proteins and broad enhancement of oxidative phosphorylation components. Functional calcium imaging demonstrates comparable neuronal dynamics across conditions but reveals robust, recurrent network-level bursts under BP, consistent with strengthened connectivity. BP also preserves retinal ganglion cells and maintains expression of canonical markers, aligning with a BDNF-centred interactome and improved circuit integration. Collectively, these findings identify the culture environment as a critical determinant of AAV efficacy in human retinal tissue models and position BP as a simple, scalable strategy to reduce vector requirements, enhance retinal cells targeting, and increase the fidelity of organoid-based non-animal testing for IRD gene therapy development.","PeriodicalId":12699,"journal":{"name":"Gene Therapy","volume":" ","pages":""},"PeriodicalIF":5.9,"publicationDate":"2026-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148897205","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0