Aptamer Technology and Its Applications in Bone Diseases

Liu, Xiangzhong; Hu, Jing; Ning, Yu; Xu, Haijia; Cai, Hantao; Yang, Aofei; Shi, Zhengshuai; Li, Zhanghua

doi:10.1177/09636897221144949

Cited by 4 publications

(1 citation statement)

References 119 publications

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“…We reported the effect of RBM-007, an RNA aptamer developed to neutralize the FGFR3 cognate ligand FGF2 (FGF2 aptamer), in in vitro and in vivo ACH models in 2021 [68]. Aptamer is a single-stranded nucleic acid molecule that binds protein with high affinity and high specificity like an antibody [69]. Aptamer has many advantages over antibodies such as strong chemical stability and rapid bulk production capability.…”

Section: Current and Emerging Drugsmentioning

confidence: 99%

Promising horizons in achondroplasia along with the development of new drugs

Ozono,

Kubota,

Michigami

2024

Endocr J

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Achondroplasia (ACH) is a representative skeletal disorder characterized by rhizomelic shortened limbs and short stature. ACH is classified as belonging to the fibroblast growth factor receptor 3 (FGFR3) group. The downstream signal transduction of FGFR3 consists of STAT1 and RAS/RAF/MEK/ERK pathways. The mutant FGFR3 found in ACH is continuously phosphorylated and activates downstream signals, resulting in abnormal proliferation and differentiation of chondrocytes in the growth plate and cranial base synchondrosis. A patient registry has been developed and has contributed to revealing the natural history of ACH patients. Concerning the short stature, the adult height of ACH patients ranges between 126.7-135.2 cm for men and 119.9-125.5 cm for women in many countries. Along with severe short stature, foramen magnum stenosis and spinal canal stenosis are major complications: the former leads to sleep apnea, breathing disorders, myelopathy, hydrocephalus, and sudden death, and the latter causes pain in the extremities, numbness, muscle weakness, movement disorders, intermittent claudication, and bladder-rectal disorders. Growth hormone treatment is available for ACH only in Japan. However, the effect of the treatment on adult height is not satisfactory. Recently, the neutral endopeptidaseresistant CNP analogue vosoritide has been approved as a new drug for ACH. Additionally in development are a tyrosine kinase inhibitor, a soluble FGFR3, an antibody against FGFR3, meclizine, and the FGF2-aptamer. New drugs will bring a brighter future for patients with ACH.

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Section: Current and Emerging Drugsmentioning

confidence: 99%

Promising horizons in achondroplasia along with the development of new drugs

Ozono,

Kubota,

Michigami

2024

Endocr J

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Magnetically induced self-assembly electrochemical biosensor with ultra-low detection limit and extended measuring range for sensitive detection of HER2 protein

Zhang,

Wang,

Liu

et al. 2024

Bioelectrochemistry

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Potential Targeting Mechanisms for Bone-Directed Therapies

Celik,

Leal,

Tomatsu

2024

IJMS

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Bone development is characterized by complex regulation mechanisms, including signal transduction and transcription factor-related pathways, glycobiological processes, cellular interactions, transportation mechanisms, and, importantly, chemical formation resulting from hydroxyapatite. Any abnormal regulation in the bone development processes causes skeletal system-related problems. To some extent, the avascularity of cartilage and bone makes drug delivery more challenging than that of soft tissues. Recent studies have implemented many novel bone-targeting approaches to overcome drawbacks. However, none of these strategies fully corrects skeletal dysfunction, particularly in growth plate-related ones. Although direct recombinant enzymes (e.g., Vimizim for Morquio, Cerezyme for Gaucher, Elaprase for Hunter, Mepsevii for Sly diseases) or hormone infusions (estrogen for osteoporosis and osteoarthritis), traditional gene delivery (e.g., direct infusion of viral or non-viral vectors with no modifications on capsid, envelope, or nanoparticles), and cell therapy strategies (healthy bone marrow or hematopoietic stem cell transplantation) partially improve bone lesions, novel delivery methods must be addressed regarding target specificity, less immunogenicity, and duration in circulation. In addition to improvements in bone delivery, potential regulation of bone development mechanisms involving receptor-regulated pathways has also been utilized. Targeted drug delivery using organic and inorganic compounds is a promising approach in mostly preclinical settings and future clinical translation. This review comprehensively summarizes the current bone-targeting strategies based on bone structure and remodeling concepts while emphasizing potential approaches for future bone-targeting systems.

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Aptamer Technology and Its Applications in Bone Diseases

Cited by 4 publications

References 119 publications

Promising horizons in achondroplasia along with the development of new drugs

Promising horizons in achondroplasia along with the development of new drugs

Magnetically induced self-assembly electrochemical biosensor with ultra-low detection limit and extended measuring range for sensitive detection of HER2 protein

Potential Targeting Mechanisms for Bone-Directed Therapies

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