In this review, we focus on recent advances in crossing the blood-brain barrier with AAV vectors. We first review the AAV serotypes that can be applied to treating CNS diseases. Recent progress in possible AAV crossing the BBB and transduction mechanisms are then summarized.

2024年9月8日 · Our experiments with hPSC-derived neurons suggest that LRP6 enhances its interacting AAV capsids’ neuronal potency at multiple levels, through more efficient BBB crossing and more efficient ...

2024年7月3日 · AAV.CAP-B10和AAV.CAP-B22是Viviana Gradinaru 团队基于M-CREATE方法在PHP.eB的基础上进行筛选开发的新型AAV变体。 AAV.CAP-B10在C57Bl/6J小鼠中能够实现静脉注射高效穿越血脑屏障在全脑表达，且靶向神经元（在感染星形胶质细胞和少突胶质细胞的能力上都弱于PHP.eB），同时避免 ...

2022年10月10日 · We report an adeno-associated virus vector with an enhanced ability to cross the blood–brain barrier in both rodents and non-human primates, and use it to develop systemic anti-tumour gene...

2024年5月17日 · 该研究开发了一种新型AAV基因治疗递送载体——BI-hTFR1，通过与人血脑屏障中高表达的人转铁蛋白受体（hTfR1）结合，能够有效穿越血脑屏障，将这种 ...

2020年11月17日 · In this review, we focus on recent advances in crossing the blood-brain barrier with AAV vectors. We first review the AAV serotypes that can be applied to treating CNS diseases. Recent progress in possible AAV crossing the BBB and transduction mechanisms are then summarized.

2、 基因治疗与糖生物学结合 文章中使用了基因治疗载体（AAV）直接提升核心 O-糖基化酶的表达，成功逆转 BBB 受损，这为基因治疗在神经系统疾病的精细化干预提供了新思路。 今后或能与其他蛋白酶、糖基化调控分子综合应用，进一步提高疗效和特异性。

2021年5月10日 · Adeno-associated virus (AAV), one of the most promising gene therapy vectors, has been shown to cross the BBB safely and is non-pathogenic in nature and therefore has been utilized for numerous diseases of the CNS.

2021年5月10日 · We discuss the BBB-crossing AAV vectors that have been developed to date and their development methods and provide a systematic research direction to develop more efficient BBB-crossing AAVs to treat CNS diseases as a gene delivery tool. The BBB is composed of cerebral capillaries and various components of the neurovascular unit.

One of the biggest challenges of effectively delivering AAV to the brain is to surmount the blood-brain barrier (BBB). Herein, we identified several potential BBB shuttle peptides that significantly enhanced AAV8 transduction in the brain after a systemic administration, the best of which was the THR peptide.