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1969 …2020

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Article
2020

Anti-Phospho-Tau Gene Therapy for Chronic Traumatic Encephalopathy

Sacramento, C. B., Sondhi, D., Rosenberg, J. B., Chen, A., Giordano, S., Pey, E., Lee, V., Stiles, K. M., Havlicek, D. F., Leopold, P. L., Kaminsky, S. M. & Crystal, R. G., Jan 2020, In : Human gene therapy. 31, 1-2, p. 57-69 13 p.

Research output: Contribution to journalArticle

Open Access
2 Citations (Scopus)

Intermittent exposure to whole cigarette smoke alters the differentiation of primary small airway epithelial cells in the air-liquid interface culture

Gindele, J. A., Kiechle, T., Benediktus, K., Birk, G., Brendel, M., Heinemann, F., Wohnhaas, C. T., LeBlanc, M., Zhang, H., Strulovici-Barel, Y., Crystal, R. G., Thomas, M. J., Stierstorfer, B., Quast, K. & Schymeinsky, J., 1 Dec 2020, In : Scientific reports. 10, 1, 6257.

Research output: Contribution to journalArticle

Open Access

My Pathway to Gene Therapy

Crystal, R. G., Mar 2020, In : Human gene therapy. 31, 5-6, p. 273-282 10 p.

Research output: Contribution to journalArticle

Symmetric Age Association of Retinal Degeneration in Patients with CLN2-Associated Batten Disease

Kovacs, K. D., Patel, S., Orlin, A., Kim, K., Van Everen, S., Conner, T., Sondhi, D., Kaminsky, S. M., D'Amico, D. J., Crystal, R. G. & Kiss, S., 1 Jan 2020, (Accepted/In press) In : Ophthalmology Retina.

Research output: Contribution to journalArticle

Open Access

Systemic Adeno-Associated Virus-Mediated Gene Therapy Prevents the Multiorgan Disorders Associated with Aldehyde Dehydrogenase 2 Deficiency and Chronic Ethanol Ingestion

Matsumura, Y., Li, N., Alwaseem, H., Pagovich, O. E., Crystal, R. G., Greenblatt, M. B. & Stiles, K. M., Feb 2020, In : Human gene therapy. 31, 3-4, p. 163-182 20 p.

Research output: Contribution to journalArticle

Open Access
1 Citation (Scopus)
2019

Characterization of an immortalized human small airway basal stem/progenitor cell line with airway region-specific differentiation capacity

Wang, G., Lou, H. H., Salit, J., Leopold, P. L., Driscoll, S., Schymeinsky, J., Quast, K., Visvanathan, S., Fine, J. S., Thomas, M. J. & Crystal, R., 23 Aug 2019, In : Respiratory Research. 20, 1, 196.

Research output: Contribution to journalArticle

Open Access
2 Citations (Scopus)

Exaggerated BMP4 signalling alters human airway basal progenitor cell differentiation to cigarette smoking-related phenotypes

Zuo, W. L., Yang, J., Strulovici-Barel, Y., Salit, J., Rostami, M., Mezey, J. G., O'Beirne, S. L., Kaner, R. J. & Crystal, R., 1 May 2019, In : The European respiratory journal. 53, 5

Research output: Contribution to journalArticle

Open Access
2 Citations (Scopus)

Gene Therapy Correction of Aldehyde Dehydrogenase 2 Deficiency

Matsumura, Y., Stiles, K. M., Reid, J., Frenk, E. Z., Cronin, S., Pagovich, O. E. & Crystal, R., 13 Dec 2019, In : Molecular Therapy - Methods and Clinical Development. 15, p. 72-82 11 p.

Research output: Contribution to journalArticle

Open Access
2 Citations (Scopus)

Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema

Qiu, T., Chiuchiolo, M. J., Whaley, A. S., Russo, A. R., Sondhi, D., Kaminsky, S. M., Crystal, R. & Pagovich, O. E., 1 Jan 2019, In : Allergy: European Journal of Allergy and Clinical Immunology.

Research output: Contribution to journalArticle

15 Citations (Scopus)

Point of Care Exome Sequencing Reveals Allelic and Phenotypic Heterogeneity Underlying Mendelian disease in Qatar

Fakhro, K. A., Robay, A., Rodrigues-Flores, J. L., Mezey, J. G., Al-Shakaki, A. A., Chidiac, O., Stadler, D., Malek, J. A., Imam, A. B., Sheikh, A., Azzam, A., Janahi, I., Khanjar, I., Osman, K., Ziki, M. A., Mahmah, M. A., Selim, M., Numeiri, N., Ali, R., Lakhani, S. & 3 others, Butt, F., Omran, T. B. & Crystal, R. G., 1 Dec 2019, In : Human molecular genetics. 28, 23, p. 3970-3981 12 p.

Research output: Contribution to journalArticle

Role of KRAS in regulating normal human airway basal cell differentiation

Ogawa, F., Walters, M. S., Shafquat, A., O'Beirne, S. L., Kaner, R. J., Mezey, J. G., Zhang, H., Leopold, P. L. & Crystal, R., 9 Aug 2019, In : Respiratory Research. 20, 1, 181.

Research output: Contribution to journalArticle

Open Access

Whole-methylome analysis of circulating monocytes in acute diabetic Charcot foot reveals differentially methylated genes involved in the formation of osteoclasts

Pasquier, J., Spurgeon, M., Bradic, M., Thomas, B., Robay, A., Chidiac, O., DIb, M. J., Turjoman, R., Liberska, A., Staudt, M., Fakhro, K. A. M. A., Menzies, R., Jayyousi, A., Zirie, M., Suwaidi, J. A., Malik, R., Talal, T., Tabrizi, A. R., Mezey, J., Rodriguez-Flores, J. & 2 others, Crystal, R. & Abi Khalil, C., 1 Feb 2019, In : Epigenomics. 11, 3, p. 281-296 16 p.

Research output: Contribution to journalArticle

1 Citation (Scopus)
2018

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

Rosenberg, J. B., Kaplitt, M. G., De, B. P., Chen, A., Flagiello, T., Salami, C., Pey, E., Zhao, L., Ricart Arbona, R. J., Monette, S., Dyke, J. P., Ballon, D. J., Kaminsky, S. M., Sondhi, D., Petsko, G. A., Paul, S. M. & Crystal, R., 1 Mar 2018, In : Human Gene Therapy Clinical Development. 29, 1, p. 24-47 24 p.

Research output: Contribution to journalArticle

14 Citations (Scopus)

Altered lung biology of healthy never smokers following acute inhalation of E-cigarettes

Staudt, M. R., Salit, J., Kaner, R. J., Hollmann, C. & Crystal, R., 14 May 2018, In : Respiratory Research. 19, 1, 78.

Research output: Contribution to journalArticle

21 Citations (Scopus)

Ambient pollution-related reprogramming of the human small airway epithelial transcriptome

O’Beirne, S. L., Shenoy, S. A., Salit, J., Strulovici-Barel, Y., Kaner, R. J., Visvanathan, S., Fine, J. S., Mezey, J. G. & Crystal, R., 1 Dec 2018, In : American Journal of Respiratory and Critical Care Medicine. 198, 11, p. 1413-1422 10 p.

Research output: Contribution to journalArticle

4 Citations (Scopus)

Association of thrombocytosis with COPD morbidity: The SPIROMICS and COPDGene cohorts

and for the SPIROMICS and COPDGene Investigators, 26 Jan 2018, In : Respiratory Research. 19, 1, 20.

Research output: Contribution to journalArticle

2 Citations (Scopus)

A systematic review on the genetics of male infertility in the era of next-generation sequencing

Robay, A., Abbasi, S., Akil, A., El-Bardisi, H., Arafa, M., Crystal, R. & Fakhro, K. A. M. A., 1 Mar 2018, In : Arab Journal of Urology. 16, 1, p. 53-64 12 p.

Research output: Contribution to journalArticle

5 Citations (Scopus)

Attenuation of the Niemann-Pick type C2 disease phenotype by intracisternal administration of an AAVrh.10 vector expressing Npc2

Markmann, S., J. Christie-Reid, J., Rosenberg, J. B., De, B. P., Kaminsky, S. M., Crystal, R. & Sondhi, D., 1 Aug 2018, In : Experimental Neurology. 306, p. 22-33 12 p.

Research output: Contribution to journalArticle

5 Citations (Scopus)

Corneal confocal microscopy: Neurologic disease biomarker in Friedreich ataxia

Pagovich, O. E., Vo, M. L., Zhao, Z. Z., Petropoulos, I. N., Yuan, M., Lertsuwanroj, B., Ciralsky, J., Lai, E., Kiss, S., D'Amico, D. J., Mezey, J. G., Malik, R. & Crystal, R., 1 Dec 2018, In : Annals of Neurology. 84, 6, p. 893-904 12 p.

Research output: Contribution to journalArticle

5 Citations (Scopus)

Disease characteristics and progression in patients with late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease: an observational cohort study

Nickel, M., Simonati, A., Jacoby, D., Lezius, S., Kilian, D., Van de Graaf, B., Pagovich, O. E., Kosofsky, B., Yohay, K., Downs, M., Slasor, P., Aiayi, T., Crystal, R., Kohlschütter, A., Sondhi, D. & Schulz, A., 1 Jan 2018, (Accepted/In press) In : The Lancet Child and Adolescent Health.

Research output: Contribution to journalArticle

17 Citations (Scopus)

Exome sequencing-based identification of novel type 2 diabetes risk allele loci in the Qatari population

O’Beirne, S. L., Salit, J., Rodriguez-Flores, J. L., Staudt, M. R., Khalil, C. A., Fakhro, K. A., Robay, A., Ramstetter, M. D., Malek, J. A., Zirie, M., Jayyousi, A., Badii, R., Al-Marri, A. A. N., Bener, A., Mahmoud, M., Chiuchiolo, M. J., Al-Shakaki, A., Chidiac, O., Stadler, D., Mezey, J. G. & 1 others, Crystal, R. G., Sep 2018, In : PloS one. 13, 9, e0199837.

Research output: Contribution to journalArticle

1 Citation (Scopus)

In Vivo Potency Assay for Adeno-Associated Virus-Based Gene Therapy Vectors Using AAVrh.10 as an Example

De, B. P., Chen, A., Salami, C. O., Van De Graaf, B., Rosenberg, J. B., Pagovich, O. E., Sondhi, D., Crystal, R. & Kaminsky, S. M., 1 Jun 2018, In : Human gene therapy methods. 29, 3, p. 146-155 10 p.

Research output: Contribution to journalArticle

3 Citations (Scopus)

Mandatory role of HMGA1 in human airway epithelial normal differentiation and post-injury regeneration

Zhang, H., Yang, J., Walters, M. S., Staudt, M. R., Strulovici-Barel, Y., Salit, J., Mezey, J. G., Leopold, P. L. & Crystal, R., 1 Jan 2018, In : Oncotarget. 9, 18, p. 14324-14337 14 p.

Research output: Contribution to journalArticle

2 Citations (Scopus)

Ontogeny and biology of human small airway epithelial club cells

Zuo, W. L., Shenoy, S. A., Li, S., O’Beirne, S. L., Strulovici-Barel, Y., Leopold, P. L., Wang, G., Staudt, M. R., Walters, M. S., Mason, C., Kaner, R. J., Mezey, J. G. & Crystal, R., 1 Dec 2018, In : American Journal of Respiratory and Critical Care Medicine. 198, 11, p. 1375-1388 14 p.

Research output: Contribution to journalArticle

14 Citations (Scopus)

p63 Silencing induces reprogramming of cardiac fibroblasts into cardiomyocyte-like cells

Patel, V., Singh, V. P., Pinnamaneni, J. P., Sanagasetti, D., Olive, J., Mathison, M., Cooney, A., Flores, E. R., Crystal, R., Yang, J. & Rosengart, T. K., 1 Jan 2018, (Accepted/In press) In : Journal of Thoracic and Cardiovascular Surgery.

Research output: Contribution to journalArticle

3 Citations (Scopus)

Point-of-care whole-exome sequencing of idiopathic male infertility

Fakhro, K. A. M. A., Elbardisi, H., Arafa, M., Robay, A., Rodriguez-Flores, J. L., Al-Shakaki, A., Syed, N., Mezey, J. G., Abi Khalil, C., Malek, J., Al-Ansari, A., Al Said, S. & Crystal, R., 1 Nov 2018, In : Genetics in Medicine. 20, 11, p. 1365-1373 9 p.

Research output: Contribution to journalArticle

9 Citations (Scopus)

Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease)

Sindelar, M., Dyke, J. P., Deeb, R. S., Sondhi, D., Kaminsky, S. M., Kosofsky, B. E., Ballon, D. J., Crystal, R. & Gross, S. S., 1 Dec 2018, In : Scientific Reports. 8, 1, 15229.

Research output: Contribution to journalArticle

3 Citations (Scopus)
14 Citations (Scopus)
2017

An independent component analysis confounding factor correction framework for identifying broad impact expression quantitative trait loci

Ju, J. H., Shenoy, S. A., Crystal, R. & Mezey, J. G., 1 May 2017, In : PLoS Computational Biology. 13, 5, e1005537.

Research output: Contribution to journalArticle

4 Citations (Scopus)

EGF-Amphiregulin Interplay in Airway Stem/Progenitor Cells Links the Pathogenesis of Smoking-Induced Lesions in the Human Airway Epithelium

Zuo, W. L., Yang, J., Gomi, K., Chao, I. W., Crystal, R. & Shaykhiev, R., 1 Mar 2017, In : Stem Cells. 35, 3, p. 824-837 14 p.

Research output: Contribution to journalArticle

17 Citations (Scopus)

Endothelial Cell Mediated Promotion of Ciliated Cell Differentiation of Human Airway Basal Cells via Insulin and Insulin-Like Growth Factor 1 Receptor Mediated Signaling

Gomi, K., Tang, Y., Arbelaez, V., Crystal, R. & Walters, M. S., 1 Apr 2017, In : Stem Cell Reviews and Reports. 13, 2, p. 309-317 9 p.

Research output: Contribution to journalArticle

3 Citations (Scopus)

Frequency of exacerbations in patients with chronic obstructive pulmonary disease: an analysis of the SPIROMICS cohort

Han, M. L. K., Quibrera, P. M., Carretta, E. E., Barr, R. G., Bleecker, E. R., Bowler, R. P., Cooper, C. B., Comellas, A., Couper, D. J., Curtis, J. L., Criner, G., Dransfield, M. T., Hansel, N. N., Hoffman, E. A., Kanner, R. E., Krishnan, J. A., Martinez, C. H., Pirozzi, C. B., O'Neal, W. K., Rennard, S. & 27 others, Tashkin, D. P., Wedzicha, J. A., Woodruff, P., Paine, R., Martinez, F. J., Alexis, N. E., Anderson, W. H., Boucher, R. C., Christenson, S. A., Criner, G. J., Crystal, R. G., Doerschuk, C. M., Freeman, C. M., Hastie, A. T., Kaner, R. J., Kleerup, E. C., LaVange, L. M., Lazarus, S. C., Meyers, D. A., Newell, J. D., Oelsner, E. C., Putcha, N., Rennard, S. I., Scholand, M. B., Wells, J. M., Wise, R. A. & Woodruff, P. G., Aug 2017, In : The Lancet Respiratory Medicine. 5, 8, p. 619-626 8 p.

Research output: Contribution to journalArticle

83 Citations (Scopus)

HIV Reprograms Human Airway Basal Stem/Progenitor Cells to Acquire a Tissue-Destructive Phenotype

Chung, N. P. Y., Ou, X., Khan, K. M. F., Salit, J., Kaner, R. J. & Crystal, R., 9 May 2017, In : Cell Reports. 19, 6, p. 1091-1100 10 p.

Research output: Contribution to journalArticle

5 Citations (Scopus)

In situ reprogramming to transdifferentiate fibroblasts into cardiomyocytes using adenoviral vectors: Implications for clinical myocardial regeneration

Mathison, M., Singh, V. P., Chiuchiolo, M. J., Sanagasetti, D., Mao, Y., Patel, V. B., Yang, J., Kaminsky, S. M., Crystal, R. & Rosengart, T. K., 1 Feb 2017, In : Journal of Thoracic and Cardiovascular Surgery. 153, 2, p. 329-339.e3

Research output: Contribution to journalArticle

12 Citations (Scopus)

Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1/2 clinical trial

Tardieu, M., Zérah, M., Gougeon, M. L., Ausseil, J., de Bournonville, S., Husson, B., Zafeiriou, D., Parenti, G., Bourget, P., Poirier, B., Furlan, V., Artaud, C., Baugnon, T., Roujeau, T., Crystal, R. G., Meyer, C., Deiva, K. & Heard, J. M., Sep 2017, In : The Lancet Neurology. 16, 9, p. 712-720 9 p.

Research output: Contribution to journalArticle

43 Citations (Scopus)

Radioiodinated Capsids Facilitate in Vivo Non-Invasive Tracking of Adeno-Associated Gene Transfer Vectors

Kothari, P., De, B. P., He, B., Chen, A., Chiuchiolo, M. J., Kim, D., Nikolopoulou, A., Amor-Coarasa, A., Dyke, J. P., Voss, H. U., Kaminsky, S. M., Foley, C. P., Vallabhajosula, S., Hu, B., DiMagno, S. G., Sondhi, D., Crystal, R., Babich, J. W. & Ballon, D., 6 Jan 2017, In : Scientific Reports. 7, 39594.

Research output: Contribution to journalArticle

3 Citations (Scopus)

Refining Current Scientific Priorities and Identifying New Scientific Gaps in HIV-Related Heart, Lung, Blood, and Sleep Research

Twigg, H. L., Crystal, R., Currier, J., Ridker, P., Berliner, N., Kiem, H. P., Rutherford, G., Zou, S., Glynn, S., Wong, R., Peprah, E., Engelgau, M., Creazzo, T., Colombini-Hatch, S. & Caler, E., 1 Sep 2017, In : AIDS Research and Human Retroviruses. 33, 9, p. 889-897 9 p.

Research output: Contribution to journalArticle

Role of OSGIN1 in mediating smoking-induced autophagy in the human airway epithelium

Wang, G., Zhou, H., Strulovici-Barel, Y., Al-Hijji, M., Ou, X., Salit, J., Walters, M. S., Staudt, M. R., Kaner, R. J. & Crystal, R., 3 Jul 2017, In : Autophagy. 13, 7, p. 1205-1220 16 p.

Research output: Contribution to journalArticle

17 Citations (Scopus)

Smoking-dependent distal-to-proximal repatterning of the adult human small airway epithelium

Yang, J., Zuo, W. L., Fukui, T., Chao, I. W., Gomi, K., Lee, B., Staudt, M. R., Kaner, R. J., Strulovici-Barel, Y., Salit, J., Crystal, R. & Shaykhiev, R., 1 Aug 2017, In : American Journal of Respiratory and Critical Care Medicine. 196, 3, p. 340-352 13 p.

Research output: Contribution to journalArticle

24 Citations (Scopus)

Waterpipe smoking induces epigenetic changes in the small airway epithelium

Walters, M. S., Salit, J., Ju, J. H., Staudt, M. R., Kaner, R. J., Rogalski, A. M., Sodeinde, T. B., Rahim, R., Strulovici-Barel, Y., Mezey, J. G., Almulla, A. M., Sattar, H., Mahmoud, M. & Crystal, R., 1 Mar 2017, In : PLoS One. 12, 3, e0171112.

Research output: Contribution to journalArticle

13 Citations (Scopus)
2016

Anti-epidermal growth factor receptor gene therapy for glioblastoma

Hicks, M. J., Chiuchiolo, M. J., Ballon, D., Dyke, J. P., Aronowitz, E., Funato, K., Tabar, V., Havlicek, D., Fan, F., Sondhi, D., Kaminsky, S. M. & Crystal, R., 1 Oct 2016, In : PLoS One. 11, 10, e0162978.

Research output: Contribution to journalArticle

11 Citations (Scopus)

Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy

Pagovich, O. E., Wang, B., Chiuchiolo, M. J., Kaminsky, S. M., Sondhi, D., Jose, C. L., Price, C. C., Brooks, S. F., Mezey, J. G. & Crystal, R., 1 Dec 2016, In : Journal of Allergy and Clinical Immunology. 138, 6, p. 1652-1662.e7

Research output: Contribution to journalArticle

13 Citations (Scopus)

Brain region-specific degeneration with disease progression in late infantile neuronal ceroid lipofuscinosis (CLN2 Disease)

Dyke, J. P., Sondhi, D., Voss, H. U., Yohay, K., Hollmann, C., Mancenido, D., Kaminsky, S. M., Heier, L. A., Rudser, K. D., Kosofsky, B., Casey, B. J., Crystal, R. & Ballon, D., 1 Jun 2016, In : American Journal of Neuroradiology. 37, 6, p. 1160-1169 10 p.

Research output: Contribution to journalArticle

5 Citations (Scopus)

Common Genetic Polymorphisms Influence Blood Biomarker Measurements in COPD

SPIROMICS Research Group & COPDGene Investigators, Aug 2016, In : PLoS genetics. 12, 8, e1006011.

Research output: Contribution to journalArticle

26 Citations (Scopus)

Efficacy of an adenovirus-based anti-cocaine vaccine to reduce cocaine self-administration and reacqusition using a choice procedure in rhesus macaques

Evans, S. M., Foltin, R. W., Hicks, M. J., Rosenberg, J. B., De, B. P., Janda, K. D., Kaminsky, S. M. & Crystal, R., 1 Nov 2016, In : Pharmacology Biochemistry and Behavior. 150-151, p. 76-86 11 p.

Research output: Contribution to journalArticle

12 Citations (Scopus)

Gene therapy for alpha-1 antitrypsin deficiency lung disease

Chiuchiolo, M. J. & Crystal, R., 1 Aug 2016, In : Annals of the American Thoracic Society. 13, p. S352-S369

Research output: Contribution to journalArticle

13 Citations (Scopus)

Indigenous Arabs are descendants of the earliest split from ancient Eurasian populations

Rodriguez-Flores, J. L., Fakhro, K., Agosto-Perez, F., Ramstetter, M. D., Arbiza, L., Vincent, T. L., Robay, A., Malek, J. A., Suhre, K., Chouchane, L., Badii, R., Al-Marri, A. A. N., Khalil, C. A., Zirie, M., Jayyousi, A., Salit, J., Keinan, A., Clark, A. G., Crystal, R. G. & Mezey, J. G., Feb 2016, In : Genome Research. 26, 2, p. 151-162 12 p.

Research output: Contribution to journalArticle

36 Citations (Scopus)

Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models

Zhao, L., Gottesdiener, A. J., Parmar, M., Li, M., Kaminsky, S. M., Chiuchiolo, M. J., Sondhi, D., Sullivan, P. M., Holtzman, D. M., Crystal, R. G. & Paul, S. M., 1 Aug 2016, In : Neurobiology of Aging. 44, p. 159-172 14 p.

Research output: Contribution to journalArticle

15 Citations (Scopus)

JAG1-Mediated Notch Signaling Regulates Secretory Cell Differentiation of the Human Airway Epithelium

Gomi, K., Staudt, M. R., Salit, J., Kaner, R. J., Heldrich, J., Rogalski, A. M., Arbelaez, V., Crystal, R. & Walters, M. S., 1 Aug 2016, In : Stem Cell Reviews and Reports. 12, 4, p. 454-463 10 p.

Research output: Contribution to journalArticle

7 Citations (Scopus)

Persistence of circulating endothelial microparticles in COPD despite smoking cessation

Strulovici-Barel, Y., Staudt, M. R., Krause, A., Gordon, C., Tilley, A. E., Harvey, B. G., Kaner, R. J., Hollmann, C., Mezey, J. G., Bitter, H., Pillai, S. G., Hilton, H., Wolff, G., Stevenson, C. S., Visvanathan, S., Fine, J. S. & Crystal, R. G., 1 Dec 2016, In : Thorax. 71, 12, p. 1137-1144 8 p.

Research output: Contribution to journalArticle

21 Citations (Scopus)