• 65820 Citations
  • 132 h-Index
1969 …2020

Research output per year

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Research Output

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
1 Citation (Scopus)

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
2019

Advances in the treatment of neuronal ceroid lipofuscinosis

Rosenberg, J. B., Chen, A., Kaminsky, S. M., Crystal, R. G. & Sondhi, D., 2 Nov 2019, In : Expert Opinion on Orphan Drugs. 7, 11, p. 473-500 28 p.

Research output: Contribution to journalReview article

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

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
1 Citation (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

13 Citations (Scopus)

Idiopathic and autoimmune interstitial lung disease

Gelbman, B. & Crystal, R. G., 1 Jan 2019, The Autoimmune Diseases. Elsevier, p. 1335-1354 20 p.

Research output: Chapter in Book/Report/Conference proceedingChapter

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

12 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

13 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

2 Citations (Scopus)

Biology of the Adrenal Gland Cortex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders

Markmann, S., De, B. P., Reid, J., Jose, C. L., Rosenberg, J. B., Leopold, P. L., Kaminsky, S. M., Sondhi, D., Pagovich, O. & Crystal, R., 1 Apr 2018, In : Human Gene Therapy. 29, 4, p. 403-412 10 p.

Research output: Contribution to journalReview article

7 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

14 Citations (Scopus)

Enzyme linked immunoassay (elisa) for connective tissue proteins: Type 1 collagen

Rennard, S. I., Martin, G. R. & Crystal, R., 1 Jan 2018, Immunochemistry of the Extracellular Matrix: Volume 1: Methods. CRC Press, p. 237-252 16 p.

Research output: Chapter in Book/Report/Conference proceedingChapter

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., Abi Khalil, C., Fakhro, K. A. M. A., Robay, A., Ramstetter, M. D., Malek, J., 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., 1 Sep 2018, In : PLoS One. 13, 9, e0199837.

Research output: Contribution to journalArticle

1 Citation (Scopus)

Intrapleural gene therapy for alpha-1 antitrypsin deficiency-related lung disease

Stiles, K. M., Sondhi, D., Kaminsky, S. M., De, B. P., Rosenberg, J. B. & Crystal, R., 1 Jan 2018, In : Chronic Obstructive Pulmonary Diseases. 5, 4, p. 244-257 14 p.

Research output: Contribution to journalReview article

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

11 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

7 Citations (Scopus)

THE JEREMIAH METZGER LECTURE NOVEL THERAPEUTIC STRATEGIES OF ALLERGIC AND IMMUNOLOGIC DISORDERS

Crystal, R. & Pagovich, O. E., 1 Jan 2018, In : Transactions of the American Clinical and Climatological Association. 129, p. 250-265 16 p.

Research output: Contribution to journalReview article

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)
12 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

3 Citations (Scopus)
1 Citation (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. & 45 others, Tashkin, D. P., Wedzicha, J. A., Woodruff, P., Paine, R., Martinez, F. J., Alexis, N. E., Anderson, W. H., Barr, R. G., Bleecker, E. R., Boucher, R. C., Bowler, R. P., Carretta, E. E., Christenson, S. A., Comellas, A. P., Cooper, C. B., Couper, D. J., Criner, G. J., Crystal, R. G., Curtis, J. L., Doerschuk, C. M., Dransfield, M. T., Freeman, C. M., Han, M. L. K., Hansel, N. N., Hastie, A. T., Hoffman, E. A., Kaner, R. J., Kanner, R. E., Kleerup, E. C., Krishnan, J. A., LaVange, L. M., Lazarus, S. C., Martinez, F. J., Meyers, D. A., Newell, J. D., Oelsner, E. C., O'Neal, W. K., Paine, R., Putcha, N., Rennard, S. I., Tashkin, D. P., Scholand, M. B., Wells, J. M., Wise, R. A. & Woodruff, P. G., 1 Aug 2017, In : The Lancet Respiratory Medicine. 5, 8, p. 619-626 8 p.

Research output: Contribution to journalArticle

78 Citations (Scopus)

Genetic modification of the lung directed toward treatment of human disease

Sondhi, D., Stiles, K. M., De, B. P. & Crystal, R., 1 Jan 2017, In : Human Gene Therapy. 28, 1, p. 3-84 82 p.

Research output: Contribution to journalReview article

10 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., Meyer, C., Deiva, K. & Heard, J. M., 1 Sep 2017, In : The Lancet Neurology. 16, 9, p. 712-720 9 p.

Research output: Contribution to journalArticle

39 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

2 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

16 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

22 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

12 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

10 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

12 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

4 Citations (Scopus)

Cigarette smoking induces changes in airway epithelial expression of genes associated with monogenic lung disorders

Tilley, A. E., Staudt, M. R., Salit, J., Van De Graaf, B., Strulovici-Barel, Y., Kaner, R. J., Vincent, T., Agosto-Perez, F., Mezey, J. G., Raby, B. A. & Crystal, R., 15 Jan 2016, In : American Journal of Respiratory and Critical Care Medicine. 193, 2, p. 215-217 3 p.

Research output: Contribution to journalLetter

3 Citations (Scopus)