Article
Cell Biology
Esmee Botman, Melissa S. A. M. Bevers, Caroline E. Wyers, Bert van Rietbergen, Bernd P. Teunissen, Pieter G. Raijmakers, Jan Coen Netelenbos, Joop P. van den Bergh, Elisabeth M. W. Eekhoff
Summary: In this study, high-resolution peripheral quantitative computed tomography (HR-pQCT) was used to non-invasively assess mature heterotopic ossification (HO) in two fibrodysplasia ossificans progressiva (FOP) patients. The findings suggest that isolated HO may be more microarchitecturally comparable to reference tibia data, and HO and skeletal bone can merge into one entity when contiguous.
FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY
(2021)
Article
Genetics & Heredity
Daniele De Brasi, Francesca Orlando, Valeria Gaeta, Maria De Liso, Fabio Acquaviva, Luigi Martemucci, Augusto Mastrominico, Maja Di Rocco
Summary: FOP is an ultra-rare genetic condition characterized by extraskeletal bone formation, with activating mutations of the ACVR1 gene being responsible for the skeletal and nonskeletal features. The clinical phenotype includes congenital bilateral hallux valgus malformation and early-onset heterotopic ossification, with essential care interventions focusing on avoiding unnecessary surgeries and administering medications to control inflammation.
Editorial Material
Medicine, General & Internal
Mohammed Mostafa Kotb, Usama Farghaly Omar, Arun-Kumar Kaliya-Perumal
Summary: This report presents the radiological findings of an 18-year-old female with FOP, showing severe spinal and right-upper-limb abnormalities. Extensive heterotopic bone formation resulted in restricted mobility and poor quality of life. While there is no definitive treatment, preventing injuries and minimizing iatrogenic harm are crucial, and ongoing research may hold the key to a future cure for FOP.
Article
Multidisciplinary Sciences
Yeon-Suk Yang, Jung-Min Kim, Jun Xie, Sachin Chaugule, Chujiao Lin, Hong Ma, Edward Hsiao, Jaehyoung Hong, Hyonho Chun, Eileen M. Shore, Frederick S. Kaplan, Guangping Gao, Jae-Hyuck Shim
Summary: The study developed gene therapy approaches that demonstrated efficacy in mouse models and human induced pluripotent stem cells, showing potential for preventing disabling heterotopic ossification in fibrodysplasia ossificans progressiva. These promising treatments could offer significant clinical benefits for patients with this rare genetic disorder.
NATURE COMMUNICATIONS
(2022)
Article
Endocrinology & Metabolism
Haitao Wang, Qiang Zhang, Frederick S. Kaplan, Robert J. Pignolo
Summary: Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease caused by mutations in ACVR1/ALK2, leading to soft tissue injuries resulting in muscles transforming into ectopic bone, preventing normal movement.
JOURNAL OF BONE AND MINERAL RESEARCH
(2022)
Article
Endocrinology & Metabolism
Haitao Wang, Carmen L. De Cunto, Robert J. Pignolo, Frederick S. Kaplan
Summary: Progressive heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP) exhibits distinctive spatial and temporal patterns, correlating with infrared thermographs of the human body. Dysregulated temperature response of connective tissue progenitor cells in FOP patients influences the anatomical distribution of HO initiation and sustainment at various sites. This provides insights into potential therapeutic targets for this disabling condition.
Article
Endocrinology & Metabolism
Frederick S. Kaplan, David T. Teachey, Jeffrey R. Andolina, David M. Siegel, Edna E. Mancilla, Edward C. Hsiao, Mona Al Mukaddam, David M. Rocke, Robert J. Pignolo
Summary: This study reported improvements in symptoms in three children with FOP treated with imatinib, supporting the design of further clinical trials.
Review
Anatomy & Morphology
Oscar Will Towler, Eileen M. Shore
Summary: FOP is an ultra-rare genetic disease caused by ACVR1 gene mutation that leads to increased BMP pathway signaling. Apart from extra-skeletal bone formation, the mutation also affects skeletal bone development, resulting in bilateral malformation of the great toes and other skeletal deformities in individuals with FOP.
DEVELOPMENTAL DYNAMICS
(2022)
Article
Pediatrics
Ambika Gupta, Puneeta Mishra, Madhumita Roy Chowdhury, Shah Alam Khan, Manisha Jana, Madhulika Kabra, Neerja Gupta
Summary: This study evaluates the natural history of Fibrodysplasia Ossificans Progressiva (FOP) and highlights possible masqueraders causing diagnostic delay and iatrogenic interventions. The findings indicate that clinical suspicion followed by molecular testing is a straightforward and effective method for confirming FOP. Diagnostic delay may result in disease progression and unnecessary invasive interventions.
INDIAN JOURNAL OF PEDIATRICS
(2023)
Review
Biotechnology & Applied Microbiology
Elisabeth M. W. Eekhoff, Ruben D. de Ruiter, Bernard J. Smilde, Ton Schoenmaker, Teun J. de Vries, Coen Netelenbos, Edward C. Hsiao, Christiaan Scott, Nobuhiko Haga, Zvi Grunwald, Carmen L. De Cunto, Maja di Rocco, Patricia L. R. Delai, Robert J. Diecidue, Vrisha Madhuri, Tae-Joon Cho, Rolf Morhart, Clive S. Friedman, Michael Zasloff, Gerard Pals, Jae-Hyuck Shim, Guangping Gao, Frederick Kaplan, Robert J. Pignolo, Dimitra Micha
Summary: Fibrodysplasia ossificans progressiva (FOP) is a rare and devastating genetic disease where soft connective tissue turns into heterotopic bone. Although the genetic defect is known, effective treatments have been hindered by the complexity of the disease. Gene therapy shows promise as a therapeutic option for FOP, but the role of the immune system and the primary causative cell remain unknown.
HUMAN GENE THERAPY
(2022)
Article
Medicine, Research & Experimental
John B. Lees-Shepard, Sean J. Stoessel, Julian T. Chandler, Keith Bouchard, Patricia Bento, Lorraine N. Apuzzo, Parvathi M. Devarakonda, Jeffrey W. Hunter, David J. Goldhamer
Summary: Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease characterized by progressive and catastrophic heterotopic ossification (HO). This study found that a monoclonal blocking antibody, JAB0505, exacerbated injury-induced HO in FOP mouse models, raising concerns about its safety and efficacy for treating FOP patients.
JOURNAL OF CLINICAL INVESTIGATION
(2022)
Editorial Material
Biochemistry & Molecular Biology
Roberto Ravazzolo
Summary: The Special Issue on Fibrodysplasia Ossificans Progressiva has published a collection of articles that provide valuable insights into the disease mechanism and potential therapeutic approaches for this rare genetic disorder.
Article
Medicine, Research & Experimental
Senem Aykul, Lily Huang, Lili Wang, Nanditha M. Das, Sandra Reisman, Yonaton Ray, Qian Zhang, Nyanza Rothman, Kalyan C. Nannuru, Vishal Kamat, Susannah Brydges, Luca Troncone, Laura Johnsen, Paul B. Yu, Sergio Fazio, John Lees-Shepard, Kevin Schutz, Andrew J. Murphy, Aris N. Economides, Vincent Idone, Sarah J. Hatsell
Summary: Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder characterized by progressive and cumulative heterotopic ossification (HO) of skeletal muscles, ligaments, tendons, and fascia. It is caused by mutations in the ACVR1 gene, enabling ACVR1 to utilize activin A as an agonistic ligand. However, our findings suggest that anti-ACVR1 antibodies should not be considered as therapeutics for FOP, as they stimulate ossification and activate signaling of FOP-mutant ACVR1 in vivo.
JOURNAL OF CLINICAL INVESTIGATION
(2022)
Review
Biochemistry & Molecular Biology
Fatima Khan, Xiaobing Yu, Edward C. Hsiao
Summary: Fibrodysplasia Ossificans Progressiva (FOP) is an ultra-rare disorder characterized by heterotopic ossifications at extraskeletal sites. It is caused by gain-of-function mutations in the Acvr1/Alk2 gene, leading to severe cardiopulmonary and neurological dysfunctions.
Article
Endocrinology & Metabolism
Sarah E. Warner, Frederick S. Kaplan, Robert J. Pignolo, Stacy E. Smith, Edward C. Hsiao, Carmen De Cunto, Maja Di Rocco, Kathleen Harnett, Donna Grogan, Harry K. Genant
Summary: In this study, low-dose whole-body computed tomography (WBCT) was found to be more sensitive and less prone to positional ambiguity compared to dual energy X-ray absorptiometry (DXA) for measuring heterotopic ossification (HO) in patients with Fibrodysplasia ossificans progressiva (FOP). Based on these findings, low-dose WBCT was chosen as the preferred imaging method for assessing total body burden of HO in patients with FOP.
CALCIFIED TISSUE INTERNATIONAL
(2021)
Article
Polymer Science
Antonina Simakova, Saadyah Averick, Arman Moini Jazani, Krzysztof Matyjaszewski
Summary: This article reports a method for preparing cationic nanogels by atom transfer radical polymerization under inverse microemulsion conditions. The use of a hydrophilic inimer prevents the growth of droplets during polymerization. The surface functionality of the nanogels can be modulated by applying different reactive surfactants or grafting hydrophilic shells. The synthesized cationic nanogels are biocompatible and have a high complexation ability for plasmid DNA.
MACROMOLECULAR CHEMISTRY AND PHYSICS
(2023)
Article
Chemistry, Multidisciplinary
Kriti Kapil, Grzegorz Szczepaniak, Michael R. R. Martinez, Hironobu Murata, Arman Moini Jazani, Jaepil Jeong, Subha R. R. Das, Krzysztof Matyjaszewski
Summary: In this study, hyperbranched polymethacrylates were synthesized by green-light-induced atom transfer radical polymerization (ATRP) under biologically relevant conditions. By using a water-soluble SBA inibramer as a inducer during the copolymerization of methacrylate monomers, well-defined branched polymethacrylates can be obtained in less than 30 minutes with predetermined molecular weights, tunable degree of branching, and low dispersity values (1.14 <= D <= 1.33). Moreover, SBA inibramer enables the synthesis of bioconjugates with a well-controlled branched architecture.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
(2023)
Review
Chemistry, Multidisciplinary
Jing Lyu, Yinghao Li, Zishan Li, Piotr Polanowski, Jeremiasz K. Jeszka, Krzysztof Matyjaszewski, Wenxin Wang
Summary: This article presents a minireview on the most important modelling techniques and their applications in free radical polymerization (FRP) and reversible-deactivation radical polymerization (RDRP) of multivinyl monomers (MVMs). The combination of experiments and modelling can provide valuable assets for polymer researchers, inspiring new thinking for future MVMs experiment optimization and product design.
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
(2023)
Article
Chemistry, Multidisciplinary
Julian Sobieski, Illia Ruzhylo, Grzegorz Szczepaniak, Adam Gorczynski, Krzysztof Matyjaszewski, Eric Manoury, Rinaldo Poli
Summary: Simple synthetic routes for the regioselective deuteration of tris[2-(dimethylamino)ethyl]amine (Me6TREN) variants are described. Formation of imine with formaldehyde-d(2) from tris(2-aminoethyl)amine (TREN) and subsequent reduction with NaBD4 resulted in N[CH2CH2N(CD3)(2)](3) or d(18)-Me6TREN in 79% yield. A trisubstitution protocol from 2-bromo-N,N-dimethylacetamide and ammonium carbonate, followed by reduction of the N(CH2CONMe2)(3) intermediate with lithium aluminum deuteride, afforded N[CH2CD2N(CH3)(2)](3) or (d(6)-arm)-Me6TREN in three steps and 52% overall yield. A similar protocol using 2-bromo-N,N-dimethyl-d(2)-acetamide, obtained from d(4)-acetic acid in two steps, and reduction of the N(CD2CONMe2)(3) intermediate with lithium aluminum hydride, resulted in N[CD2CH2N(CH3)(2)](3) or (d(6)-cap)-Me6TREN in four steps and 13% overall yield.
Article
Polymer Science
Dirk J. Schild, Juliana Bem, Grzegorz Szczepaniak, Arman Moini Jazani, Krzysztof Matyjaszewski
Summary: We report a photoinduced initiators for continuous activator regeneration atom transfer radical polymerization (PICAR ATRP) using sodium pyruvate and blue light (λ(max) = 456 nm). Water-soluble oligo(ethylene oxide) methyl ether methacrylate (OEOMA(500)) was polymerized under biologically relevant conditions. Well-defined polymers with high molecular weights and low dispersity were synthesized in less than 60 min using Cu/Fe dual catalysis and without deoxygenation.
JOURNAL OF POLYMER SCIENCE
(2023)
Article
Chemistry, Multidisciplinary
Mateusz Olszewski, Xiaolei Hu, Ting-Chih Lin, Krzysztof Matyjaszewski, Natalia Lebedeva, Philip Taylor
Summary: Star polymers were synthesized via ARGET ATRP and used as effective stabilizers for Pickering emulsions. The interfacial properties of PEO stars adsorbed at oil-water interfaces were investigated using interfacial tension and interfacial rheology. The results offer important insights into the interfacial rheology of PEO star polymers in the context of their application as stabilizers for Pickering emulsions.
Article
Polymer Science
Kriti Kapil, Arman Moini Jazani, Grzegorz Szczepaniak, Hironobu Murata, Mateusz Olszewski, Krzysztof Matyjaszewski
Summary: Photoinduced ATRP techniques using green light and dual catalysis have been developed for efficient and controlled polymerization. This method allows rapid synthesis of well-defined polymers without the need for deoxygenation, overcoming the limitations of UV light-induced polymerization. The system also enables temporal control and synthesis of various hydrophilic acrylate monomers.
Article
Polymer Science
Guanyi Hou, Yingxue Hao, Ruifeng Jiang, Sai Li, Yonglai Lu, Jun Liu, Liqun Zhang, Krzysztof Matyjaszewski
Summary: The rapidly developing aerospace industry has a severe requirement for rubber components utilized for spacecraft, especially the resistance to extreme temperature differences because of the complex space environment. However, it is difficult to increase the service temperature range of the existing elastomeric materials based on traditional methods. By introducing a new type of long-side chain siloxane, a novel silicone elastomer with an unprecedented wide temperature range (-149-400°C) is designed, which does not introduce other heteroatoms.
Article
Chemistry, Multidisciplinary
Kostas Parkatzidis, Nghia P. Truong, Krzysztof Matyjaszewski, Athina Anastasaki
Summary: This paper introduces a photocatalytic ATRP depolymerization method, which significantly reduces the reaction temperature while enabling temporal regulation and high monomer recovery through the use of low-toxicity catalysts and visible light irradiation. The depolymerization rate can be modulated by simply turning the light on and off, and the method is compatible with various polymers and light sources while preserving the end-group fidelity.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
(2023)
Article
Chemistry, Multidisciplinary
Jaepil Jeong, Xiaolei Hu, Hironobu Murata, Grzegorz Szczepaniak, Marta Rachwalak, Anna Kietrys, Subha R. R. Das, Krzysztof Matyjaszewski
Summary: Combining synthetic polymers with RNA allows for creating RNA-based materials with unique functions. By developing an acylation reagent, we achieved the direct incorporation of an atom transfer radical polymerization (ATRP) initiator into both synthetic oligoribonucleotides and natural biomass RNA. This approach enables the grafting of polymer chains from RNA and the synthesis of RNA-polymer hybrids with narrow molecular weight distributions, including RNA bottlebrushes, hydrogels, and stimuli-responsive materials.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
(2023)
Article
Polymer Science
Martin Cvek, Arman Moini Jazani, Julian Sobieski, Thaiskang Jamatia, Krzysztof Matyjaszewski
Summary: ZnO nanocrystals were successfully synthesized and used as a heterogeneous co-catalyst in mechano- and photoATRP. The photoATRP with ZnO exhibited faster reaction kinetics compared to mechanoATRP and did not require excess ligand. This study demonstrated the versatility and robustness of ZnO-cocatalyzed photoATRP for various monomers.
Article
Polymer Science
Julian Sobieski, Sajjad Dadashi Silab, Lucas Thevenin, Krzysztof Matyjaszewski, Christophe Fliedel, Rinaldo Poli
Summary: The termination processes of the tertiary carbomethoxyisopropylradical, (COOMe)Me2C & BULL;, were investigated by H-1 NMR. The investigation assessed the contributions of the Cu-I/L-catalyzed radical termination (CRT) and the reductive radical termination (RRT) to the spontaneous bimolecular radical terminations (RT). It also reassessed the product distribution for the RT of the diffused and caged radicals obtained from the thermal activation of V-601.
Article
Polymer Science
Rongguan Yin, Yuqi Zhao, Jaepil Jeong, Jirameth Tarnsangpradit, Tong Liu, So Young An, Yue Zhai, Xiaolei Hu, Michael R. Bockstaller, Krzysztof Matyjaszewski
Summary: Gradient copolymer brush nanoparticles with different compositional ratios were synthesized from surface-modified silica nanoparticles, and they demonstrated better mechanical properties and uniform microstructures.
Article
Chemistry, Multidisciplinary
Izabela Zaborniak, Martyna Korbecka, Zuzanna Michno, Pawel Chmielarz, Krzysztof Matyjaszewski
Summary: In this study, a high molecular weight water-soluble polymer was synthesized through polymerization in a vegetable oil-based inverse emulsion, resulting in low dispersity and high molecular weight. This approach offers advantages in terms of environmental friendliness and can replace toxic organic solvents.
ACS SUSTAINABLE CHEMISTRY & ENGINEERING
(2023)
Article
Chemistry, Multidisciplinary
Kostas Parkatzidis, Nghia P. Truong, Krzysztof Matyjaszewski, Athina Anastasaki
Summary: A photocatalytic ATRP depolymerization method is introduced, which lowers the reaction temperature and enables temporal control, achieving high monomer recovery and end-group fidelity.
JOURNAL OF THE AMERICAN CHEMICAL SOCIETY
(2023)
