Targeting strategies for bone diseases: signaling pathways and clinical studies

H Xu, W Wang, X Liu, W Huang, C Zhu, Y Xu… - … and Targeted Therapy, 2023 - nature.com
Since the proposal of Paul Ehrlich's magic bullet concept over 100 years ago, tremendous
advances have occurred in targeted therapy. From the initial selective antibody, antitoxin to …

Development of AAV-mediated gene therapy approaches to treat skeletal diseases

C Lin, MB Greenblatt, G Gao, JH Shim - Human Gene Therapy, 2024 - liebertpub.com
Adeno-associated viral (AAV) vectors have emerged as crucial tools in advancing gene
therapy for skeletal diseases, offering the potential for sustained expression with low …

The future of sickle cell disease therapeutics rests in genomics

A Wonkam - Disease models & mechanisms, 2023 - journals.biologists.com
Sickle cell disease (SCD) is the most-common monogenic recessive disease in humans,
annually affecting almost 300,000 newborns worldwide, 75% of whom live in Africa …

[HTML][HTML] AAV-Mediated Targeting of the Activin A-ACVR1R206H Signaling in Fibrodysplasia Ossificans Progressiva

YS Yang, C Lin, H Ma, J **e, FS Kaplan, G Gao… - Biomolecules, 2023 - mdpi.com
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized
by progressive disabling heterotopic ossification (HO) at extra-skeletal sites. Here, we …

Immunologic aspects in fibrodysplasia ossificans progressiva

A Diolintzi, MS Pervin, EC Hsiao - Biomolecules, 2024 - mdpi.com
Background: Inflammation is a major driver of heterotopic ossification (HO), a condition of
abnormal bone growth in a site that is not normally mineralized. Purpose of review: This …

Microfluidic one-step synthesis of a metal− organic framework for osteoarthritis therapeutic microRNAs delivery

K Yang, M Ni, C Xu, L Wang, L Han, S Lv… - … in Bioengineering and …, 2023 - frontiersin.org
As a class of short non-coding ribonucleic acids (RNAs), microRNAs (miRNA) regulate gene
expression in human cells and are expected to be nucleic acid drugs to regulate and treat a …

Chemical modification of AAV9 capsid with N-ethyl maleimide alters vector tissue tropism

PL Mulcrone, AK Lam, D Frabutt, J Zhang… - Scientific Reports, 2023 - nature.com
Although more adeno-associated virus AAV-based drugs enter the clinic, vector tissue
tropism remains an unresolved challenge that limits its full potential despite that the tissue …

Heterotopic mineralization (ossification or calcification) in aged musculoskeletal soft tissues: a new candidate marker for aging

GC Dai, H Wang, Z Ming, PP Lu, YJ Li, YC Gao… - Ageing Research …, 2024 - Elsevier
Aging can lead to various disorders in organisms and with the escalating impact of
population aging, the incidence of age-related diseases is steadily increasing. As a major …

[HTML][HTML] Navigating the complex landscape of fibrodysplasia ossificans progressiva: from current paradigms to therapeutic frontiers

S Anwar, T Yokota - Genes, 2023 - mdpi.com
Fibrodysplasia ossificans progressiva (FOP) is an enigmatic, ultra-rare genetic disorder
characterized by progressive heterotopic ossification, wherein soft connective tissues …

Oxidative phosphorylation is a pivotal therapeutic target of fibrodysplasia ossificans progressiva

L Sun, Y **, M Nishio, M Watanabe… - Life Science …, 2024 - life-science-alliance.org
Heterotopic ossification (HO) is a non-physiological bone formation where soft tissue
progenitor cells differentiate into chondrogenic cells. In fibrodysplasia ossificans progressiva …