Development of Medicines for Rare Pediatric Diseases DOI Creative Commons
Danilo Marimpietri, Guendalina Zuccari

Pharmaceuticals, Journal Year: 2023, Volume and Issue: 16(4), P. 513 - 513

Published: March 30, 2023

To date, approximately 7000 rare diseases exist, affecting between 6% and 8% of the global population >30 million people in European Union [...].

Language: Английский

miRNAs as potential game-changers in retinoblastoma: Future clinical and medicinal uses DOI
Ahmed S. Doghish, Hebatallah Ahmed Mohamed Moustafa, Mohammed S. Elballal

et al.

Pathology - Research and Practice, Journal Year: 2023, Volume and Issue: 247, P. 154537 - 154537

Published: May 19, 2023

Language: Английский

Citations

42

Exploring the Potential of Nanotechnology in Pediatric Healthcare: Advances, Challenges, and Future Directions DOI Creative Commons
Hossein Omidian, Kwadwo Mfoafo

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(6), P. 1583 - 1583

Published: May 24, 2023

The utilization of nanotechnology has brought about notable advancements in the field pediatric medicine, providing novel approaches for drug delivery, disease diagnosis, and tissue engineering. Nanotechnology involves manipulation materials at nanoscale, resulting improved effectiveness decreased toxicity. Numerous nanosystems, including nanoparticles, nanocapsules, nanotubes, have been explored their therapeutic potential addressing diseases such as HIV, leukemia, neuroblastoma. also shown promise enhancing diagnosis accuracy, availability, overcoming blood–brain barrier obstacle treating medulloblastoma. It is important to acknowledge that while offers significant opportunities, there are inherent risks limitations associated with use nanoparticles. This review provides a comprehensive summary existing literature on highlighting its revolutionize healthcare recognizing challenges need be addressed.

Language: Английский

Citations

20

Pediatric Drug Development: Reviewing Challenges and Opportunities by Tracking Innovative Therapies DOI Creative Commons
Cátia Domingues, Ivana Jarak, Francisco Veiga

et al.

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(10), P. 2431 - 2431

Published: Oct. 6, 2023

The paradigm of pediatric drug development has been evolving in a “carrot-and-stick”-based tactic to address population-specific issues. However, the off-label prescription adult medicines patients remains feature clinical practice, which may compromise age-appropriate evaluation treatments. Therefore, United States and European Pediatric Formulation Initiative have recommended applying nanotechnology-based delivery systems tackle some these challenges, particularly inorganic, polymeric, lipid-based nanoparticles. Connected with these, advanced therapy medicinal products (ATMPs) also highlighted, optimistic perspectives for population. Despite results achieved using innovative therapies, workforce that congregates and/or caregivers, healthcare stakeholders, developers, physicians continues be utmost relevance promote standardized guidelines development, enabling fast lab-to-clinical translation. taking into consideration significance this topic, work aims compile current landscape by (1) outlining historic regulatory panorama, (2) summarizing challenges formulation, (3) delineating advantages/disadvantages approaches, such as nanomedicines ATMPs pediatrics. Moreover, attention will given role pharmaceutical technologists developers conceiving medicines.

Language: Английский

Citations

16

Nanoparticle-based delivery systems as emerging therapy in retinoblastoma: recent advances, challenges and prospects DOI Creative Commons
Adaeze Linda Onugwu, Onyinyechi Lydia Ugorji, Chinasa A. Ufondu

et al.

Nanoscale Advances, Journal Year: 2023, Volume and Issue: 5(18), P. 4628 - 4648

Published: Jan. 1, 2023

Nanoparticles utilized in targeting retinoblastoma cells demonstrated improved uptake and intracellular internalization, sustained retention, excellent cytotoxicity, enhanced apoptosis antitumor activity.

Language: Английский

Citations

13

New retinoblastoma (RB) drug delivery approaches: anti‐tumor effect of atrial natriuretic peptide (ANP)‐conjugated hyaluronic‐acid‐coated gold nanoparticles for intraocular treatment of chemoresistant RB DOI Creative Commons
André Haase,

Natalia Miroschnikov,

Stefan Klein

et al.

Molecular Oncology, Journal Year: 2024, Volume and Issue: 18(4), P. 832 - 849

Published: Jan. 12, 2024

Intraocular drug delivery is a promising approach for treatment of ocular diseases. Chemotherapeutic drugs used in retinoblastoma (RB) often lead to side effects and resistances. Therefore, new adjuvant therapies are needed treat chemoresistant RBs. Biocompatible gold nanoparticles (GNPs) have unique antiangiogenic properties can inhibit cancer progression. The combination low‐molecular‐weight hyaluronan (HA) enhances the stability GNPs promotes distribution across barriers. Attached HA‐GNPs, atrial natriuretic peptide (ANP), which diminishes neovascularization eye, therapeutic agent RB treatment. In study presented, we established ANP‐coupled HA‐GNPs investigated their effect on tumor formation potential cells an ovo chicken chorioallantoic membrane model orthotopic vivo rat eye model. Treatment etoposide‐resistant with ANP‐HA‐GNPs resulted significantly reduced growth angiogenesis compared controls. antitumorigenic could be verified model, including noninvasive application form via drops. Our data suggest that represent minimally invasive, option RB.

Language: Английский

Citations

4

USP14 increases the sensitivity of retinoblastoma to cisplatin by mediating the ferroptosis DOI Creative Commons
Han Liu,

Qiang Gan,

Yongping Lai

et al.

Naunyn-Schmiedeberg s Archives of Pharmacology, Journal Year: 2024, Volume and Issue: 397(11), P. 8671 - 8680

Published: May 31, 2024

Abstract The aim of this study is to explore the function USP14 on sensitivity retinoblastoma (RB) cisplatin (DDP) and underlying mechanism. was knockdown in Y79 cells by transfecting three siRNAs (si-USP14-1, si-USP14-2, si-USP14-3), with si-USP14 NC as negative control. si-USP14-3 selected results Western blotting. CCK-8 assay used detect IC50 growth curve. cell cycle, apoptosis, ROS level were measured flow cytometry. expression P-GP, ERCC1, survivin, GPX4, FTH1, ACSL4, NOX1, COX2, FASN determined blotting assay. CO-IP utilized evaluate interaction between FASN. DDP Y79/DDP 7.83 µM 24.67 µM, respectively. Compared control groups, increased apoptotic rate level, arrested cycle S phase observed USP14-knockdown cells. G0/G1 control, extremely downregulated or cells, accompanied elevated ACSL4. identified according result By silencing levels resistance-related proteins (P-GP, survivin), ferroptosis-related (FTH1 GPX4), lipid metabolism-related (NOX1, FASN) dramatically reduced, enhanced restrained DNA content, indicating that might suppress resistance RB mediating ferroptosis, which an important target for treating RB.

Language: Английский

Citations

4

LIFU (Low-Intensity Focused Ultrasound) Activated Tumor-Starvation/Oxidative-Stress Combined Therapy for Treating Retinoblastoma DOI Creative Commons
Luya Quan, Mengzhu Wang, Zhigang Wang

et al.

International Journal of Nanomedicine, Journal Year: 2025, Volume and Issue: Volume 20, P. 4085 - 4103

Published: April 1, 2025

To overcome the limitations of traditional therapies in treating retinoblastoma, like low efficiency, systematic toxicity and poor biocompatibility. PPFG (PLGA-PFH-Fe3O4-GOx) nanoparticles were synthesized by ultrasound double emulsification method characterized dynamic laser scattering, ultraviolet spectrometry, confocal scanning microscopy (CLSM), transmission electron (TEM) (SEM). Phase transition low-intensity focused (LIFU) was observed microscope imaging. Cellular uptake compared between Y79 HUVEC cells. ROS production detected 2',7'-dichlorofluorescin diacetate (DCFH-DA). Cell apoptosis flow cytometry. In vivo therapeutic effects verified tumor volume, HE staining, TUNEL PCNA staining. The bio-safety serum biochemistry. NPs possesses good stability, biocompatibility tumor-preferred uptake, with a core-shell spherical structure an average size 255.6nm which increases to over 100μm under LIFU irradiation. utilized as stimuli, undergoes sequential reaction starting phase PFH causing release oxygen carried GOx/SPIO NPs, followed supplemented facilitating enzymatic activity glucose consumption GOx cells (tumor starvation), H2O2 produced during can further participate SPIO NPs-mediated Fenton (CDT), generating massive ROS. continuously generated together cut down nutrients effectively inhibited progression tumors, synergistically enhanced starvation promoted cell ultimately kills tumour No off-site injuries other major organs. this study, conduct LIFU-triggered combinational therapy on basis cascade among PFH, treat retinoblastoma vitro/vivo. It showed great potentials combating retinoblastoma.

Language: Английский

Citations

0

Bioactive Polymeric Nanoparticles of Moringa oleifera Induced Phyto-Photothermal Sensitization for the Enhanced Therapy of Retinoblastoma DOI Creative Commons
Sushma V. Mudigunda, Deepak B. Pemmaraju,

Sri Amruthaa Sankaranarayanan

et al.

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(2), P. 475 - 475

Published: Jan. 31, 2023

Treatment of retinoblastoma is limited due to its delayed detection and inaccesbility drugs reach the retina crossing blood-retinal barrier. With advancements in nanotechnology, photothermal therapy (PTT) employing plasmonic nanomaterials and/or NIR dyes have emerged as an affordable alternative owing spatial control that offered by modality leading localized enhanced therapeutic efficacy with minimal invasiveness. However, clinical application increased heat shock resistance tumor cells response generated via PTT. Hence, this study, we explore role novel biomolecular fraction Moringa oleifera (DFM) encapsulated within a polymeric nanosystem, for anti-heat protein (HSP) activity. The MO extract was co-encapsulated sensitizing dye, IR820 into biodegradable polycaprolactone (PCL) nano-delivery system (PMIR NPs). transduction PMIR NPs validated vitro against cell lines. inherent fluorescence DFM utilized evaluate cellular internalization using microscopy flow cytometry. overall oxidative damage downregulation HSP70 expression upon treatment laser irradiation evaluated densiometric analysis Western blotting. Overall, exhibited excellent anti-cancer activity when combined PTT downregulated cells.

Language: Английский

Citations

9

Nanoparticle-mediated gene therapy as a novel strategy for the treatment of retinoblastoma DOI
Madhurima Mandal, Indranil Banerjee, Mahitosh Mandal

et al.

Colloids and Surfaces B Biointerfaces, Journal Year: 2022, Volume and Issue: 220, P. 112899 - 112899

Published: Oct. 4, 2022

Language: Английский

Citations

13

Cannabinoid-Based Ocular Therapies and Formulations DOI Creative Commons
Sofia M. Saraiva, Lucía Martín‐Banderas, Matilde Durán‐Lobato

et al.

Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(4), P. 1077 - 1077

Published: March 27, 2023

The interest in the pharmacological applications of cannabinoids is largely increasing a wide range medical areas. Recently, research on its potential role eye conditions, many which are chronic and/or disabling and need new alternative treatments, has intensified. However, due to cannabinoids’ unfavorable physicochemical properties adverse systemic effects, along with ocular biological barriers local drug administration, delivery systems needed. Hence, this review focused following: (i) identifying disease conditions potentially subject treatment their role, emphasis glaucoma, uveitis, diabetic retinopathy, keratitis prevention Pseudomonas aeruginosa infections; (ii) reviewing formulations that must be controlled optimized for successful administration; (iii) analyzing works evaluating cannabinoid-based results limitations; (iv) could useful administration strategies. Finally, an overview current advances limitations field, technological challenges overcome prospective further developments, provided.

Language: Английский

Citations

7