CRISPR-Cpf1 system and its applications in animal genome editing DOI

Yawei Han,

Zisen Jia,

Keli Xu

и другие.

Molecular Genetics and Genomics, Год журнала: 2024, Номер 299(1)

Опубликована: Авг. 1, 2024

Язык: Английский

Alzheimer’s disease: Insights and new prospects in disease pathophysiology, biomarkers and disease-modifying drugs DOI Creative Commons
Ana Rita Monteiro, Daniel José Barbosa, Fernando Remião

и другие.

Biochemical Pharmacology, Год журнала: 2023, Номер 211, С. 115522 - 115522

Опубликована: Март 28, 2023

Alzheimer's disease (AD) is one of the most prevalent neurodegenerative diseases that affect millions people worldwide, with both prevalence and incidence increasing age. It characterized by cognitive decline associated, specifically, degeneration cholinergic neurons. The problem this even more fundamental as available therapies remain fairly limited mainly focused on symptoms' relief. Although aetiology remains elusive, two main pathological hallmarks are described: i) presence neurofibrillary tangles formed unfolded protein aggregates (hyperphosphorylated Tau protein) ii) extracellular amyloid-beta peptide. Given complexity surrounding pathogenesis disease, several potential targets have been highlighted interrelated upon its progression, such oxidative stress accumulation metal ions. Thus, advances made development innovative multitarget therapeutical compounds to delay progression restore cell function. This review focuses ongoing research new insights emerging disease-modifying drugs for AD treatment. Furthermore, classical novel biomarkers early diagnosis their role in assisting improvement targeted will also be approached.

Язык: Английский

Процитировано

134

CRISPR/Cas9 Mediated Therapeutic Approach in Huntington’s Disease DOI Open Access
Süleyman Serdar Alkanlı, Nevra Alkanlı, Arzu Ay

и другие.

Molecular Neurobiology, Год журнала: 2022, Номер 60(3), С. 1486 - 1498

Опубликована: Дек. 9, 2022

Язык: Английский

Процитировано

46

Stem cell therapeutics and gene therapy for neurologic disorders DOI Creative Commons
Kevin S. Chen, Emily J. Koubek,

Stacey A. Sakowski

и другие.

Neurotherapeutics, Год журнала: 2024, Номер 21(4), С. e00427 - e00427

Опубликована: Июль 1, 2024

Язык: Английский

Процитировано

10

Advancements in Surgical Therapies for Drug-Resistant Epilepsy: A Paradigm Shift towards Precision Care DOI Creative Commons
Zhong Chen, Kang Yang,

Nianhua Wang

и другие.

Neurology and Therapy, Год журнала: 2025, Номер unknown

Опубликована: Фев. 10, 2025

Epilepsy, a prevalent neurological disorder characterized by recurrent seizures, affects millions worldwide, with significant proportion resistant to pharmacological treatments. Surgical interventions have emerged as pivotal in managing drug-resistant epilepsy (DRE), aiming reduce seizure frequency or achieve freedom. Traditional resective surgeries evolved technological advances, enhancing precision and safety. Neurostimulation techniques, such responsive neurostimulation (RNS) deep brain stimulation (DBS), now provide personalized, real-time management, offering alternatives traditional surgery. Minimally invasive ablative methods, laser interstitial thermal therapy (LITT) Magnetic Resonance-guided Focused Ultrasound (MRgFUS), allow for targeted destruction of epileptogenic tissue reduced risks faster recovery times. The use stereo-electroencephalography (SEEG) robotic assistance has further refined surgical precision, outcomes. These advancements mark paradigm shift towards medicine care, promising improved management quality life patients globally. This review outlines the latest innovations surgery, emphasizing their mechanisms clinical implications improve outcomes DRE.

Язык: Английский

Процитировано

2

Revisiting Alpha-Synuclein Pathways to Inflammation DOI Open Access
Patrícia Lyra, Vanessa Machado, Silvia Rota

и другие.

International Journal of Molecular Sciences, Год журнала: 2023, Номер 24(8), С. 7137 - 7137

Опубликована: Апрель 12, 2023

Alpha-synuclein (α-Syn) is a short presynaptic protein with an active role on synaptic vesicle traffic and the neurotransmitter release reuptake cycle. The α-Syn pathology intertwines formation of Lewy Bodies (multiprotein intraneuronal aggregations), which, combined inflammatory events, define various α-synucleinopathies, such as Parkinson’s Disease (PD). In this review, we summarize current knowledge mechanistic pathways to inflammation, well eventual microbial dysbiosis α-Syn. Furthermore, explore possible influence mitigation conclusion, given rising burden neurodegenerative disorders, it pressing clarify pathophysiological processes underlying in order consider existing low-grade chronic states potential pathway toward management prevention conditions, aim starting search for concrete clinical recommendations particular population.

Язык: Английский

Процитировано

16

Advances in stem cell and other therapies for Huntington’s disease: An update DOI Creative Commons

L. A. Conner,

Bhairavi Srinageshwar,

Bakke Jl

и другие.

Brain Research Bulletin, Год журнала: 2023, Номер 199, С. 110673 - 110673

Опубликована: Май 29, 2023

Huntington's disease (HD) is a neurodegenerative disorder caused by an autosomal dominant mutation leading to abnormal CAG repeat expansion. The result the synthesis of toxic misfolded protein, called mutant huntingtin protein (mHTT). Most current treatments are palliative, but latest research has expanded into multiple modalities, including stem cells, gene therapy, and even use 3D cell structures, organoids. Stem as treatment for HD included various types such mesenchymal neural embryonic reprogrammed cells induced pluripotent cells. goal been develop transplant grafts that will replace existing mutated neurons, well release trophic factors neuronal support. Additionally, in modification using CRISPR-Cas9, PRIME editing, other forms genetic modifications continuing evolve. recently, advancements modeling have yielded tissue models, These organoids offer unique opportunity structured graft which, ideally, models normal human brain more accurately. This manuscript summarizes recent modifications, potential HD.

Язык: Английский

Процитировано

12

Exploring the Potential and Challenges of CRISPR Delivery and Therapeutics for Genetic Disease Treatment DOI Creative Commons

Xinpu Yang,

Thuỳ Anh Bùi, Haoqi Mei

и другие.

Advanced Functional Materials, Год журнала: 2024, Номер 34(38)

Опубликована: Май 27, 2024

Abstract Human genetic disorders, arising from a range of irregularities, can significantly affect human physiology, often with limited available treatment options. The development the CRISPR system, facilitating precise editing genome, has opened new avenues for addressing mutations found in various disorders. However, there is currently lack comprehensive reviews that specifically address application diseases. To bridge this gap, review focuses on exploring advancements technology and their utility therapeutic approaches This introduces explains fundamental mechanisms editing, highlights latest technology. Additionally, it examines three delivery techniques, including physical delivery, viral vectors, nanocarriers. It further CRISPR's applications Finally, identifies primary hurdles associated industrial ethics considerations should be addressed before medical context.

Язык: Английский

Процитировано

3

Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies DOI Creative Commons

Amrutha Banda,

Olivia Impomeni,

Aparana Singh

и другие.

Vaccines, Год журнала: 2024, Номер 12(6), С. 636 - 636

Опубликована: Июнь 7, 2024

Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR)-associated enzyme-CAS holds great promise for treating many uncured human diseases and illnesses by precisely correcting harmful point mutations disrupting disease-causing genes. The recent Food Drug Association (FDA) approval of the first CRISPR-based gene therapy sickle cell anemia marks beginning a new era in editing. However, delivering CRISPR specifically into diseased cells vivo is significant challenge an area intense research. identification CRISPR/Cas variants, particularly ultra-compact CAS systems with robust editing activities, paves way low-capacity delivery vectors to be used therapies. technology has evolved beyond DNA cover wide spectrum functionalities, including RNA targeting, disease diagnosis, transcriptional/epigenetic regulation, chromatin imaging, high-throughput screening, modeling. can engineer B-cells produce potent antibodies more effective vaccines enhance CAR T-cells precise efficient targeting tumor cells. challenges, off-target effects, toxicity, immune responses, inadequate tissue-specific delivery. Overcoming these challenges necessitates development specific system. This entails strategically utilizing gRNAs conjunction variants mitigate effects. review seeks delve intricacies mechanism, explore progress therapies, evaluate systems, highlight limitations, outline necessary precautions, scrutinize ethical considerations associated its application.

Язык: Английский

Процитировано

3

Neuroprotective potential of Epigenetic modulators, its regulation and therapeutic approaches for the management of Parkinson's disease DOI
Shobha Kumari, Sakshi Gupta,

Rajesh Sukhija

и другие.

European Journal of Pharmacology, Год журнала: 2024, Номер 985, С. 177123 - 177123

Опубликована: Ноя. 12, 2024

Язык: Английский

Процитировано

3

ALZHEIMER'S DISEASE: COMPREHENSIVE INSIGHTS INTO RISK FACTORS, BIOMARKERS, AND ADVANCED TREATMENT APPROACHES DOI Open Access
Suresh Janadri,

SHREELAXMI DADMI,

Manjunatha P. Mudagal

и другие.

International Journal of Current Pharmaceutical Research, Год журнала: 2025, Номер unknown, С. 1 - 10

Опубликована: Янв. 15, 2025

Alzheimer’s disease (AD) is a prevalent neurodegenerative disorder primarily affecting individuals over 60. It multifactorial driven by both modifiable factors, such as lifestyle, diet, and prior health conditions, well non-modifiable like age, genetics, family history. The key pathological features of AD include the buildup amyloid β plaques neurofibrillary tangles resulting from hyperphosphorylated tau proteins in brain. Biomarkers protein levels cerebrospinal fluid (CSF) blood are essential for diagnosing tracking progression. Current research focuses on developing drugs targeting multiple aspects pathology, including inflammation, oxidative stress, synaptic dysfunction, accumulation. These treatments aim to slow cognitive decline neuronal damage. Given complexity AD, multi-targeted therapeutic approaches being explored enhance treatment efficacy. This review provides an overview risk biomarkers used diagnosis, latest advances clinical drug development.

Язык: Английский

Процитировано

0