This review explores recent advancements in gene therapy as a potential treatment for neurodegenerative diseases, focusing on intervention mechanisms, administration routes, and associated limitations. Following the PRISMA procedure guidelines, we systematically analyzed studies published since 2020 using the PICO framework to derive reliable conclusions. The efficacy of various gene therapies was evaluated for Parkinson's disease (n = 12), spinal muscular atrophy (n = 8), Huntington's disease (n = 3), Alzheimer's disease (n = 3), and amyotrophic lateral sclerosis (n = 6). For each condition, we assessed the therapeutic approach, curative or disease-modifying potential, delivery methods, advantages, drawbacks, and side effects. Results indicate that gene therapies targeting specific genes are particularly effective in monogenic disorders, with promising clinical outcomes expected in the near future. In contrast, in polygenic diseases, therapies primarily aim to promote cell survival. A major challenge remains: the translation of animal model success to human clinical application. Additionally, while intracerebral delivery methods enhance therapeutic efficacy, they are highly invasive. Despite these hurdles, gene therapy represents a promising frontier in the treatment of neurodegenerative diseases, underscoring the need for continued research to refine and personalize treatments for each condition.
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http://dx.doi.org/10.3390/ijms252312485 | DOI Listing |
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC11640776 | PMC |
Biol Res
December 2024
Unidad de Innovación en Prevención y Oncología de Precisión Centro Oncológico, Facultad de Medicina, Unidad de Innovación en Prevención y Oncología de Precisión Universidad Católica del Maule, Talca, 3480094, Chile.
Background: Breast cancer is a leading cause of cancer-related mortality worldwide, with hereditary forms accounting for approximately 10% of cases. In Chile, significant gaps exist in genetic counseling and testing, particularly within the public health system. This study presents the implementation and outcomes of the first regional hereditary cancer program in the Maule region of Chile, aimed at improving detection and management of hereditary breast cancer.
View Article and Find Full Text PDFJ Nanobiotechnology
December 2024
Beijing Institute of Radiation Medicine, 27 Taiping Road, Beijing, 100850, China.
In the post-COVID-19 era, drug-resistant bacterial infections emerge as one of major death causes, where multidrug-resistant Acinetobacter baumannii (MRAB) and drug-resistant Pseudomonas aeruginosa (DRPA) represent primary pathogens. However, the classical antibiotic strategy currently faces the bottleneck of drug resistance. We develop an antimicrobial strategy that applies the selective delivery of CRISPR/Cas9 plasmids to pathogens with biomimetic cationic hybrid vesicles (BCVs), irrelevant to bacterial drug resistance.
View Article and Find Full Text PDFZhonghua Yu Fang Yi Xue Za Zhi
December 2024
Department of Laboratory Medicine, The Second Xiangya Hospital, Central South University, Changsha410011, China.
To investigate the drug-resistance mutations and treatment of hospitalized children with Mycoplasma pneumoniae pneumonia (MPP) in Hunan Province. Children with pneumonia, who were hospitalized in the pediatric ward of the Second Xiangya Hospital of Central South University from January 1, 2023, to December 31, 2023, were enrolled in this study, and their clinical data was also collected. The targeted next-generation sequencing (tNGS) was used to detect Mycoplasma pneumoniae (MP) infection and drug-resistance mutations, and the drug-resistance and treatment in children with MPP were also analyzed.
View Article and Find Full Text PDFESMO Open
December 2024
Cancer Institute and Blood Disorders, Hospital Clinic, Barcelona; Translational Genomics and Targeted Therapies in Solid Tumors, August Pi I Sunyer Biomedical Research Institute (IDIBAPS), Barcelona; Department of Medicine, University of Barcelona, Barcelona; Reveal Genomics, Barcelona; Breast Cancer Unit, IOB-QuirónSalud, Barcelona, Spain. Electronic address:
Therapies targeting human epidermal growth factor receptor 2 (HER2)-positive breast cancer significantly impact patient outcomes, quality of life, and health care systems. While chemotherapy and trastuzumab improve survival in early-stage HER2-positive breast cancer, variability in clinical and biological characteristics leads to different response to therapies and outcomes. Clinical guidelines provide general recommendations, but significant uncertainty persists in identifying an optimal treatment plan for individual patients.
View Article and Find Full Text PDFCancer Rep (Hoboken)
December 2024
Department of Obstetrics and Gynecology, School of Medicine, Jichi Medical University, Shimotsuke, Tochigi, Japan.
Background: Vasohibin-1 (VASH1), an angiogenic inhibitor, exhibits tubulin carboxypeptidase activity, which is involved in microtubule functions. Paclitaxel, the core chemotherapeutic agent for ovarian cancer chemotherapy, has a point of action on microtubules and may interact with VASH1.
Aims: To examine the influence of VASH1 on intracellular tubulin detyrosination status, cyclin B1 expression, and paclitaxel chemosensitivity using VASH1-overexpressing ovarian cancer cell lines.
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