Neurosurgical gene therapy for central nervous system diseases DOI Creative Commons
Ruchit V. Patel, Pranav Nanda, R. Mark Richardson

и другие.

Neurotherapeutics, Год журнала: 2024, Номер 21(4), С. e00434 - e00434

Опубликована: Июль 1, 2024

Viral vector mediated gene therapies for neurodegenerative and neurodevelopmental conditions that require neurosurgical administration continue to expand. We systematically reviewed the National Institutes of Health (NIH) ClinicalTrials.gov database identify all clinical trials studying in-vivo viral targeted CNS diseases. isolated studies which delivered using approaches: intracisternal, intraventricular, and/or intraparenchymal. Clinical primarily registered in international countries were included if they referenced by an NIH trial. performed a scoping review preclinical supported each human Key data aggregated characterize capsid design, delivery methods, expression profile, benefit. A total 64 identified active, completed, terminated, long-term follow-up stages. range across pediatric adult populations are being studied with therapy, including Alzheimer's disease, Parkinson's AADC deficiency, sphingolipidoses, mucopolysaccharidoses, neuronal ceroid lipofuscinoses, spinal muscular atrophy, adrenoleukodystrophy, Canavan frontotemporal dementia, Huntington's Rett syndrome, Dravet mesial temporal lobe epilepsy, glutaric acidemia. Adeno-associated vectors (AAVs) utilized majority tested therapies, serotypes, regulatory elements, monitoring varying based on disease studied. Intraparenchymal has evolved significantly, MRI-guided convection-enhanced established as gold standard method pioneering novel targets.

Язык: Английский

Drug delivery to the central nervous system DOI
Elizabeth Nance, Suzie H. Pun, Rajiv Saigal

и другие.

Nature Reviews Materials, Год журнала: 2021, Номер 7(4), С. 314 - 331

Опубликована: Дек. 3, 2021

Язык: Английский

Процитировано

220

Neuronal circuitry for recognition memory of object and place in rodent models DOI Creative Commons
Owen Y. Chao, Susanne Nikolaus, Yi Yang

и другие.

Neuroscience & Biobehavioral Reviews, Год журнала: 2022, Номер 141, С. 104855 - 104855

Опубликована: Сен. 8, 2022

Язык: Английский

Процитировано

87

Brain-wide neuronal circuit connectome of human glioblastoma DOI
Yusha Sun, Xin Wang, Daniel Y. Zhang

и другие.

Nature, Год журнала: 2025, Номер unknown

Опубликована: Янв. 16, 2025

Язык: Английский

Процитировано

4

Combining brain perturbation and neuroimaging in non-human primates DOI Creative Commons
P. Christiaan Klink, Jean‐François Aubry, Vincent P. Ferrera

и другие.

NeuroImage, Год журнала: 2021, Номер 235, С. 118017 - 118017

Опубликована: Март 29, 2021

Brain perturbation studies allow detailed causal inferences of behavioral and neural processes. Because the combination brain methods measurement techniques is inherently challenging, research in humans has predominantly focused on non-invasive, indirect perturbations, or neurological lesion studies. Non-human primates have been indispensable as a neurobiological system that highly similar to while simultaneously being more experimentally tractable, allowing visualization functional structural impact systematic perturbation. This review considers state art non-human primate with focus approaches can be combined neuroimaging. We consider both non-reversible (lesions) reversible temporary perturbations such electrical, pharmacological, optical, optogenetic, chemogenetic, pathway-selective, ultrasound based interference methods. Method-specific considerations from development community are offered facilitate this field support further innovations. conclude by identifying novel avenues for innovation highlighting clinical translational potential

Язык: Английский

Процитировано

70

Local and long-distance organization of prefrontal cortex circuits in the marmoset brain DOI Creative Commons
Akiya Watakabe, Henrik Skibbe, Ken Nakae

и другие.

Neuron, Год журнала: 2023, Номер 111(14), С. 2258 - 2273.e10

Опубликована: Май 16, 2023

The prefrontal cortex (PFC) has dramatically expanded in primates, but its organization and interactions with other brain regions are only partially understood. We performed high-resolution connectomic mapping of the marmoset PFC found two contrasting corticocortical corticostriatal projection patterns: "patchy" projections that formed many columns submillimeter scale nearby distant "diffuse" spread widely across striatum. Parcellation-free analyses revealed representations gradients these projections' local global distribution patterns. also demonstrated column-scale precision reciprocal connectivity, suggesting contains a mosaic discrete columns. Diffuse showed considerable diversity laminar patterns axonal spread. Altogether, fine-grained reveal important principles long-distance circuits marmosets provide insights into functional primate brain.

Язык: Английский

Процитировано

38

Micro/nanosystems for controllable drug delivery to the brain DOI Creative Commons

Mingzhen Tian,

Zhichao Ma, Guang‐Zhong Yang

и другие.

The Innovation, Год журнала: 2023, Номер 5(1), С. 100548 - 100548

Опубликована: Ноя. 27, 2023

•Micro/nanosystems show their potential to address the challenges of precise drug delivery brain.•Microfluidic platforms enable creation biomimetic in vitro brain models.•Micro/nano materials is emerging as a key player controllable delivery.•The minimally invasive fiberbot microsystem reduces procedure's invasiveness.•Image tracking micro/nanosystems allows for controlled therapeutic interventions. Drug crucial treatment central nervous system disorders. While significant progress has been made recent years, there are still major achieving brain. Unmet clinical needs arise from various factors, including transport, handling large doses, methods crossing biological barriers, use imaging guidance, and effective models analyzing delivery. Recent advances have shown promise addressing some these challenges. These include utilization microfluidic test validate process setting, development novel micro/nanocarriers loads across blood-brain barrier, implementation micro-intervention systems delivering drugs through intraparenchymal or peripheral routes. In this article, we present review latest developments We also delve into relevant diseases, conventional methods. addition, discuss future prospects robotic equipped with directed transportation, real-time image closed-loop control.

Язык: Английский

Процитировано

36

Characterizing and targeting glioblastoma neuron-tumor networks with retrograde tracing DOI Creative Commons
Svenja Kristin Tetzlaff, Ekin Reyhan, Nikolas Layer

и другие.

Cell, Год журнала: 2024, Номер unknown

Опубликована: Дек. 1, 2024

Glioblastomas are invasive brain tumors with high therapeutic resistance. Neuron-to-glioma synapses have been shown to promote glioblastoma progression. However, a characterization of tumor-connected neurons has hampered by lack technologies. Here, we adapted retrograde tracing using rabies viruses investigate and manipulate neuron-tumor networks. Glioblastoma rapidly integrated into neural circuits across the brain, engaging in widespread functional communication, cholinergic driving invasion. We uncovered patient-specific tumor-cell-state-dependent differences synaptogenic gene expression associated connectivity subsequent invasiveness. Importantly, radiotherapy enhanced increased neuronal activity. In turn, simultaneous activity inhibition showed effects, indicative role for neuron-to-glioma contributing Lastly, rabies-mediated genetic ablation halted progression, offering viral strategy tackle glioblastoma. Together, this study provides framework comprehensively characterize networks target

Язык: Английский

Процитировано

13

Viral Tools for Neural Circuit Tracing DOI
Qing Liu, Yang Wu,

Huadong Wang

и другие.

Neuroscience Bulletin, Год журнала: 2022, Номер 38(12), С. 1508 - 1518

Опубликована: Сен. 22, 2022

Язык: Английский

Процитировано

34

A review of HSV pathogenesis, vaccine development, and advanced applications DOI Creative Commons

Lan Bai,

Jiuzhi Xu, Linghui Zeng

и другие.

Molecular Biomedicine, Год журнала: 2024, Номер 5(1)

Опубликована: Авг. 29, 2024

Herpes simplex virus (HSV), an epidemic human pathogen threatening global public health, gains notoriety for its complex pathogenesis that encompasses lytic infection of mucosal cells, latent within neurons, and periodic reactivation. This intricate interplay, coupled with HSV's sophisticated immune evasion strategies, gives rise to various diseases, including genital lesions, neonatal encephalitis, cancer. Despite more than 70 years relentless research, effective preventive or therapeutic vaccine against HSV has yet emerge, primarily due the limited understanding virus-host interactions, which in turn impedes identification targets. However, unique pathological features, substantial genetic load capacity, high replicability, transmissibility, neurotropism, render it a promising candidate applications, spanning oncolytic virotherapy, gene therapies, even as imaging tracer neuroscience. In this review, we comprehensively update recent breakthroughs evasion, critically summarize progress made development, discuss multifaceted applications biological tool. Importantly, highlight both success challenges, emphasizing critical need intensified research into HSV, aim providing deeper insights can not only advance treatment strategies but also broaden application horizons.

Язык: Английский

Процитировано

9

Neural circuit research using molecular barcode technology DOI Creative Commons
Yasuhiro Go

Neuroscience Research, Год журнала: 2025, Номер unknown

Опубликована: Март 1, 2025

Язык: Английский

Процитировано

1