Human Stem Cell Therapy for the Cure of Type 1 Diabetes Mellitus (T1D): A Hurdle Course between Lights and Shadows DOI Creative Commons
Riccardo Calafiore, Giovanni Luca, Francesco Gaggia

и другие.

Endocrines, Год журнала: 2024, Номер 5(4), С. 465 - 477

Опубликована: Окт. 5, 2024

Background: T1D is a severe metabolic disorder due to selective autoimmune pancreatic islet β-cell killing, which results in complete abrogation of endogenous insulin secretion. The affected patients, once the disease clinically overt, must immediately undertake supplementation according intensive therapy regimens prevent onset acute and chronic complications, some them potentially lethal. Replacement destroyed β-cells with fresh vital endocrine tissue, either whole organ or isolated islets transplantation, started few decades ago progressively encouraging results, although exogenous withdrawal was obtained minor cohort treated patients. restricted availability donor organs coupled general immunosuppression treatment recipients avoid graft immune rejection may, at least partially, explain limited success achieved by these procedures. Results: introduction pluripotent stem cells (either human embryonic origin adult genetically induced pluripotency) that can be differentiated toward secretory β-like could provide an indefinite resource for insulin-producing (IPCs). Conclusions: Because use embryos may encounter ethical problems, employment multipotent mesenchymal (MSCs) extracted from several tissues represent alternative option. MSCs are associated strong immunoregulatory properties alter early stages β-cell-directed autoimmunity T1D, other than holding potential differentiate themselves into cells. Lights shadows new strategies cure their advancement state reviewed.

Язык: Английский

A Multidisciplinary Approach of Type 1 Diabetes: The Intersection of Technology, Immunotherapy, and Personalized Medicine DOI Open Access
Denisa Batîr-Marin, Claudia Simona Ștefan, Monica Boev

и другие.

Journal of Clinical Medicine, Год журнала: 2025, Номер 14(7), С. 2144 - 2144

Опубликована: Март 21, 2025

Background: Type 1 diabetes (T1D) is a chronic autoimmune disorder characterized by the destruction of pancreatic β-cells, leading to absolute insulin deficiency. Despite advancements in therapy and glucose monitoring, achieving optimal glycemic control remains challenge. Emerging technologies novel therapeutic strategies are transforming landscape T1D management, offering new opportunities for improved outcomes. Methods: This review synthesizes recent treatment, focusing on innovations continuous monitoring (CGM), automated delivery systems, smart formulations, telemedicine, artificial intelligence (AI). Additionally, we explore biomedical approaches such as stem cell therapy, gene editing, immunotherapy, gut microbiota modulation, nanomedicine-based interventions, trace element-based therapies. Results: Advances digital health, including CGM integration with hybrid closed-loop pumps AI-driven predictive analytics, have significantly real-time management. AI telemedicine enhanced personalized care patient engagement. Furthermore, regenerative medicine strategies, β-cell replacement, CRISPR-based immunomodulatory therapies, hold potential disease modification. Probiotics microbiome-targeted therapies demonstrated promising effects maintaining metabolic homeostasis, while elements provide additional regulate sensitivity oxidative stress. Conclusions: The future management shifting toward precision integrated technological solutions. While these present avenues, challenges long-term efficacy, safety, accessibility, clinical validation must be addressed. A multidisciplinary approach, combining research, intelligence, nanotechnology, will essential translate into practice, ultimately improving quality life individuals T1D.

Язык: Английский

Процитировано

0

CRISPR-Cas Systems and Genome Editing: Beginning the Era of CRISPR/Cas Therapies for Humans DOI Open Access
Dmitry S. Karpov

International Journal of Molecular Sciences, Год журнала: 2024, Номер 25(10), С. 5292 - 5292

Опубликована: Май 13, 2024

Harnessing of CRISPR/Cas (Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR-associated genes) systems for detection, chemical modification, and sequence editing nucleic acids dramatically changed many fields fundamental science, biotechnology, biomedicine [...]

Язык: Английский

Процитировано

4

Research on the application of CRISPR technology in the treatment of type 1 diabetes (T1D) DOI Creative Commons
Mingyi Chen, Juntong Liu,

L Zhang

и другие.

Theoretical and Natural Science, Год журнала: 2025, Номер 82(1), С. 19 - 26

Опубликована: Янв. 15, 2025

Diabetes is a chronic disease with rapid increase in incidence, which general problem around the world. Two main types of diabetes are included. Polygenic includes type 1 (T1D) and 2 (T2D), their monogenic forms juvenile mature (MODY) neonatal (NDM). CRISPR technology gene therapy low cost but very high feasibility. At present, researchers have envisioned use treatment T1D, this has off-target rate, immune rejection no abundant clinical trials human body to verify technology, resulting fact that not really used treatment. In middle. This article mainly analyzes summarizes problems solutions encountered by CRISPR-cas9 from basic theory idealized models. provides new for T1D also references technology. addition, such as been solved. Future research can focus on solution suppression cellular after impact re-mutation.

Язык: Английский

Процитировано

0

Islet Cell Replacement and Regeneration for Type 1 Diabetes: Current Developments and Future Prospects DOI Creative Commons
Arthur Rech Tondin, Giacomo Lanzoni

BioDrugs, Год журнала: 2025, Номер unknown

Опубликована: Фев. 7, 2025

Язык: Английский

Процитировано

0

Advancing toward a curative frontier: an updated narrative review on stem-cell therapy in pediatric type 1 diabetes DOI
Samia Aziz Sulaiman,

Abdallah Alaarag,

Nadin Rayyan

и другие.

World Journal of Pediatrics, Год журнала: 2025, Номер unknown

Опубликована: Апрель 21, 2025

Язык: Английский

Процитировано

0

Expanding Horizons of CRISPR/Cas Technology: Clinical Advancements, Therapeutic Applications, and Challenges in Gene Therapy DOI Open Access
Ahmad Bairqdar,

Polina E. Karitskaya,

Г. А. Степанов

и другие.

International Journal of Molecular Sciences, Год журнала: 2024, Номер 25(24), С. 13321 - 13321

Опубликована: Дек. 12, 2024

CRISPR–Cas technology has transformed the field of gene editing, opening new possibilities for treatment various genetic disorders. Recent years have seen a surge in clinical trials using CRISPR–Cas-based therapies. This review examines current landscape implementation trials, with data from key registries, including Australian New Zealand Clinical Trials Registry, Chinese Trial Register, and ClinicalTrials.gov. Emphasis is placed on mechanism action tested therapies, delivery method, most recent findings each trial.

Язык: Английский

Процитировано

2

Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine DOI Creative Commons

Sarkar Sardar Azeez,

Rahin Shareef Hamad,

Bahra Kakamin Hamad

и другие.

Frontiers in Genome Editing, Год журнала: 2024, Номер 6

Опубликована: Дек. 12, 2024

CRISPR-Cas (Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated proteins) has undergone marked advancements since its discovery as an adaptive immune system in bacteria and archaea, emerged a potent gene-editing tool after the successful engineering of synthetic guide RNA (sgRNA) toward targeting specific DNA sequences with high accuracy. Besides editing ability, further-developed Cas variants can also edit epigenome, rendering versatile for genome epigenome manipulation pioneering force precision medicine. This review explores latest technology therapeutic biomedical applications, highlighting transformative impact on Moreover, current status CRISPR therapeutics clinical trials is discussed. Finally, we address persisting challenges prospects technology.

Язык: Английский

Процитировано

2

CRISPR/Cas9-mediated genome editing: from basic research to gene therapy DOI Open Access
Rajeev Goel

International Journal of Research in Medical Sciences, Год журнала: 2024, Номер 12(6), С. 2200 - 2211

Опубликована: Май 31, 2024

CRISPR/Cas9 mediated genome editing is one of the most significant molecular tools discovered to edit desired genes. It has ushered in a new era novel possibilities gene therapy. system was originally observed as part adaptive immune bacteria. later on adapted carry precise and targeted alterations DNA human cells be used for therapy correct genetic disorders treat various severe diseases associated with changes. Besides this, been employed pharmacogenomics develop drugs based patient’s genes, modifying organisms research even diagnostic purposes developing CRISPR COVID-9 test. The recent approval cellular by FDA named “Casgevy” sickle cell anemia testimonial potentials innovative therapies. This review details mechanisms its utilization ongoing clinical trials treatment not only monogenic like disease, thalassemia, blindness but also treating multi-factorial cancers, cardiac diseases, diabetes, autoimmune viral infections such immunodeficiency virus (HIV) etc. An attempt made discuss limitations, challenges ethical frameworks encompassing settings.

Язык: Английский

Процитировано

1

Flash glucose monitoring in diabetes DOI Creative Commons
Annel Lameijer

Опубликована: Июнь 11, 2024

Adequate and timely glucose level assessment is of utmost importance for persons with diabetes when aiming optimal glycemic control thus avoid or delay the development micro- macrovascular complications. In this thesis, effects Flash Glucose Monitoring (FGM) on well-being in are explored. FGM a form continuous monitoring that requires scanning sensor to obtain information aboutthe concentration interstitial fluid. contrast fingerprick (point) measurements, more convenient, provides insight fluctuations trends during day night, allows frequent assessment. Previous studies had limited follow-up period were mainly focused change HbA1c as outcome parameter. first part longer-term effectiveness who started was assessed. continued use two years, sustained improvement quality life observed. Commencement also associated mental well-being. A higher baseline correlated greater decline HbA1c.The second thesis into changes parameters different levels (dys)regulation. Initiation parameters, irrespective pre-use regulation treatment modality. flash scan rate control, e.g. time target range, hyperglycemia variability.

Язык: Английский

Процитировано

0

Advances in CRISPR/Cas systems-based cell and gene therapy DOI

Arpita Poddar,

Farah Ahmady,

Prashanth Prithviraj

и другие.

Progress in molecular biology and translational science, Год журнала: 2024, Номер unknown, С. 161 - 183

Опубликована: Янв. 1, 2024

Язык: Английский

Процитировано

0