• 65527 Citations
  • 132 h-Index
1969 …2019
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Research Output 1969 2019

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Review article
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

Neuronal Ceroid-Lipofuscinoses
Therapeutics
Biomarkers
Animal Models
Enzyme Replacement Therapy
2018
7 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

Congenital Adrenal Hyperplasia
Dependovirus
Adrenal Cortex
Adrenal Glands
Steroid 21-Hydroxylase

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

alpha 1-Antitrypsin Deficiency
alpha 1-Antitrypsin
Genetic Therapy
Lung Diseases
Leukocyte Elastase

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

Genetic Therapy
Peanut Hypersensitivity
Hereditary Angioedemas
Immune System Diseases
Monoclonal Antibodies
2017
10 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

Lung
Therapeutics
Architectural Accessibility
Inborn Genetic Diseases
Respiratory Syncytial Viruses
2016
13 Citations (Scopus)

Gene therapy for metachromatic leukodystrophy

Rosenberg, J. B., Kaminsky, S. M., Aubourg, P., Crystal, R. & Sondhi, D., 1 Nov 2016, In : Journal of Neuroscience Research. 94, 11, p. 1169-1179 11 p.

Research output: Contribution to journalReview article

Metachromatic Leukodystrophy
Genetic Therapy
Globoid Cell Leukodystrophy
Cerebroside-Sulfatase
Enzyme Replacement Therapy
2014
92 Citations (Scopus)

Adenovirus: The first effective in vivo gene delivery vector

Crystal, R., 1 Jan 2014, In : Human Gene Therapy. 25, 1, p. 3-11 9 p.

Research output: Contribution to journalReview article

Gene Transfer Techniques
Genetic Vectors
Adenoviridae
Genetic Therapy
Genes
34 Citations (Scopus)

Airway basal cells: The "smoking gun" of chronic obstructive pulmonary disease

Crystal, R., 1 Jan 2014, In : American Journal of Respiratory and Critical Care Medicine. 190, 12, p. 1355-1362 8 p.

Research output: Contribution to journalReview article

Chronic Obstructive Pulmonary Disease
Smoking
Lung
Cell Biology
Stem Cells
10 Citations (Scopus)

Gene therapy: Charting a future course - Summary of a national institutes of health workshop, April 12, 2013

O'Reilly, M., Federoff, H. J., Fong, Y., Kohn, D. B., Patterson, A. P., Ahmed, N., Asokan, A., Boye, S. E., Crystal, R., De Oliveira, S., Gargiulo, L., Harper, S. Q., Ikeda, Y., Jambou, R., Montgomery, M., Prograis, L., Rosenthal, E., Sterman, D. H., Vandenberghe, L. H., Zoloth, L. & 15 others, Abedi, M., Adair, J., Adusumilli, P. S., Goins, W. F., Gray, J., Monahan, P., Popplewell, L., Sena-Esteves, M., Tannous, B., Weber, T., Wierda, W., Gopal-Srivastava, R., McDonald, C. L., Rosenblum, D. & Corrigan-Curay, J., 1 Jun 2014, In : Human Gene Therapy. 25, 6, p. 488-497 10 p.

Research output: Contribution to journalReview article

National Institutes of Health (U.S.)
Genetic Therapy
Education
Health Resources
Biotechnology
2013
4 Citations (Scopus)

Advances in the treatment of neuronal ceroid lipofuscinosis

Sondhi, D., Rosenberg, J. B., Van De Graaf, B. G., Kaminsky, S. M. & Crystal, R., 1 Jan 2013, In : Expert Opinion on Orphan Drugs. 1, 12, p. 951-975 25 p.

Research output: Contribution to journalReview article

Neuronal Ceroid-Lipofuscinoses
Enzyme Replacement Therapy
Diet Therapy
Therapeutics
Cell- and Tissue-Based Therapy
24 Citations (Scopus)

Alpha-1 antitrypsin augmentation therapy

Wewers, M. D. & Crystal, R., 1 Mar 2013, In : COPD: Journal of Chronic Obstructive Pulmonary Disease. 10, S1, p. 64-67 4 p.

Research output: Contribution to journalReview article

alpha 1-Antitrypsin
alpha 1-Antitrypsin Deficiency
Leukocyte Elastase
Inborn Genetic Diseases
Therapeutics
14 Citations (Scopus)

Gene therapy for rare diseases: Summary of a National Institutes of Health Workshop, September 13, 2012

O'Reilly, M., Kohn, D. B., Bartlett, J., Benson, J., Brooks, P. J., Byrne, B. J., Camozzi, C., Cornetta, K., Crystal, R., Fong, Y., Gargiulo, L., Gopal-Srivastava, R., High, K. A., Jacobson, S. G., Jambou, R. C., Montgomery, M., Rosenthal, E., Samulski, R. J., Skarlatos, S. I., Sorrentino, B. & 3 others, Wilson, J. M., Xie, Y. & Corrigan-Curay, J., 1 Apr 2013, In : Human Gene Therapy. 24, 4, p. 355-362 8 p.

Research output: Contribution to journalReview article

National Institutes of Health (U.S.)
Rare Diseases
Genetic Therapy
Education
Information Dissemination
16 Citations (Scopus)

Gene therapy to stimulate angiogenesis to treat diffuse coronary artery disease

Kaminsky, S. M., Rosengart, T. K., Rosenberg, J., Chiuchiolo, M. J., Van De Graaf, B., Sondhi, D. & Crystal, R., 1 Nov 2013, In : Human Gene Therapy. 24, 11, p. 948-963 16 p.

Research output: Contribution to journalReview article

Genetic Therapy
Coronary Artery Disease
Hepatocyte Growth Factor
Fibroblast Growth Factors
Angiogenesis Inducing Agents
38 Citations (Scopus)

Innate immunity and chronic obstructive pulmonary disease: A mini-review

Shaykhiev, R. & Crystal, R., 1 Oct 2013, In : Gerontology. 59, 6, p. 481-489 9 p.

Research output: Contribution to journalReview article

Innate Immunity
Chronic Obstructive Pulmonary Disease
Disease Progression
Immune System
Smoking
2012
608 Citations (Scopus)

A call for transparent reporting to optimize the predictive value of preclinical research

Landis, S. C., Amara, S. G., Asadullah, K., Austin, C. P., Blumenstein, R., Bradley, E. W., Crystal, R., Darnell, R. B., Ferrante, R. J., Fillit, H., Finkelstein, R., Fisher, M., Gendelman, H. E., Golub, R. M., Goudreau, J. L., Gross, R. A., Gubitz, A. K., Hesterlee, S. E., Howells, D. W., Huguenard, J. & 16 others, Kelner, K., Koroshetz, W., Krainc, D., Lazic, S. E., Levine, M. S., MacLeod, M. R., McCall, J. M., Iii, R. T. M., Narasimhan, K., Noble, L. J., Perrin, S., Porter, J. D., Steward, O., Unger, E., Utz, U. & Silberberg, S. D., 11 Oct 2012, In : Nature. 490, 7419, p. 187-191 5 p.

Research output: Contribution to journalReview article

Research
Research Personnel
National Institute of Neurological Disorders and Stroke
Organized Financing
Quality Improvement
8 Citations (Scopus)

Cardiac biointerventions: Whatever happened to stem cell and gene therapy?

Rosengart, T. K., Fallon, E. & Crystal, R., 1 May 2012, In : Innovations: Technology and Techniques in Cardiothoracic and Vascular Surgery. 7, 3, p. 173-179 7 p.

Research output: Contribution to journalReview article

Cell- and Tissue-Based Therapy
Genetic Therapy
Stem Cells
Heart Failure
Myocardial Ischemia
2009
59 Citations (Scopus)

Genetic Control of Wayward Pluripotent Stem Cells and Their Progeny after Transplantation

Kiuru, M., Boyer, J. L., O'Connor, T. P. & Crystal, R., 3 Apr 2009, In : Cell Stem Cell. 4, 4, p. 289-300 12 p.

Research output: Contribution to journalReview article

Pluripotent Stem Cells
Transplantation
Neoplastic Stem Cells
Transplants
Therapeutics
2008
11 Citations (Scopus)

Progress and prospects: Gene therapy for performance and appearance enhancement

Kiuru, M. & Crystal, R., 1 Mar 2008, In : Gene Therapy. 15, 5, p. 329-337 9 p.

Research output: Contribution to journalReview article

Genetic Therapy
Genetic Enhancement
Cognition
Technology
Weights and Measures
2007
42 Citations (Scopus)

Human embryonic stem cells and gene therapy

Strulovici, Y., Leopold, P. L., O'Connor, T. P., Pergolizzi, R. G. & Crystal, R., 1 May 2007, In : Molecular Therapy. 15, 5, p. 850-866 17 p.

Research output: Contribution to journalReview article

Cell- and Tissue-Based Therapy
Genetic Therapy
Genes
Human Embryonic Stem Cells
Technology
75 Citations (Scopus)

Intracellular trafficking of adenovirus: Many means to many ends

Leopold, P. L. & Crystal, R., 10 Aug 2007, In : Advanced Drug Delivery Reviews. 59, 8, p. 810-821 12 p.

Research output: Contribution to journalReview article

Adenoviridae
Capsid
Genome
Adenoviridae Infections
Nuclear Pore
2006
114 Citations (Scopus)

Genetic medicines: Treatment strategies for hereditary disorders

O'Connor, T. P. & Crystal, R., 1 Apr 2006, In : Nature Reviews Genetics. 7, 4, p. 261-276 16 p.

Research output: Contribution to journalReview article

Adult Stem Cells
Medical Genetics
Embryonic Stem Cells
Transfer RNA
Genetic Therapy
4 Citations (Scopus)

Genetic medicine strategies to protect against bioterrorism.

Boyer, J. L. & Crystal, R., 1 Dec 2006, In : Transactions of the American Clinical and Climatological Association. 117

Research output: Contribution to journalReview article

Anthrax Vaccines
Bioterrorism
Genetic Techniques
Anthrax
Genetic Vectors
2005
42 Citations (Scopus)

Adenovirus-based genetic vaccines for biodefense

Boyer, J. L., Kobinger, G., Wilson, J. M. & Crystal, R., 1 Feb 2005, In : Human Gene Therapy. 16, 2, p. 157-168 12 p.

Research output: Contribution to journalReview article

Adenoviridae
Vaccines
Transgenes
Technology
Genes
27 Citations (Scopus)

Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders

Arkin, L. M., Sondhi, D., Worgall, S., Suh, L. H. K., Hackett, N. R., Kaminsky, S. M., Hosain, S. A., Souweidane, M. M., Kaplitt, M. G., Dyke, J. P., Heier, L. A., Ballon, D. J., Shungu, D. C., Wisniewski, K. E., Greenwald, B. M., Hollmann, C. & Crystal, R., 1 Sep 2005, In : Human Gene Therapy. 16, 9, p. 1028-1036 9 p.

Research output: Contribution to journalReview article

Therapeutic Misconception
Research Personnel
Medicine
Neuronal Ceroid-Lipofuscinoses
Lysosomal Storage Diseases
5 Citations (Scopus)

Intrapleural 'outside-in' gene therapy: Therapeutics for organs of the chest via gene transfer to the pleura

Heguy, A. & Crystal, R., 1 Oct 2005, In : Current Opinion in Molecular Therapeutics. 7, 5, p. 440-453 14 p.

Research output: Contribution to journalReview article

Pleura
Genetic Therapy
Thorax
Genes
Therapeutics
2004
90 Citations (Scopus)

Administration of a replication-deficient adeno-associated virus gene transfer vector expressing the human CLN2 cDNA to the brain of children with late infantile neuronal ceroid lipofuscinosis

Crystal, R., Sondhi, D., Hackett, N. R., Kaminsky, S. M., Worgall, S., Stieg, P., Souweidane, M., Hosain, S., Heier, L., Ballon, D., Dinner, M., Wisniewski, K., Kaplitt, M., Greenwald, B. M., Howell, J. D., Strybing, K., Dyke, J. & Voss, H., 1 Nov 2004, In : Human Gene Therapy. 15, 11, p. 1131-1154 24 p.

Research output: Contribution to journalReview article

Neuronal Ceroid-Lipofuscinoses
Dependovirus
Complementary DNA
Brain
Genes
16 Citations (Scopus)

Genetic medicine at the RNA level: Modifications of the genetic repertoire for therapeutic purposes by pre-mRNA trans-splicing

Pergolizzi, R. G. & Crystal, R., 1 Aug 2004, In : Comptes Rendus - Biologies. 327, 8, p. 695-709 15 p.

Research output: Contribution to journalReview article

Trans-Splicing
trans-splicing
RNA Precursors
genetic engineering
Medicine
43 Citations (Scopus)

The bronchopulmonary pathology of alpha-1 antitrypsin (AAT) deficiency: Findings of the Death Review Committee of the national registry for individuals with severe deficiency of alpha-1 antitrypsin

Tomashefski, J. F., Crystal, R., Wiedemann, H. P., Mascha, E. & Stoller, J. K., 1 Dec 2004, In : Human Pathology. 35, 12, p. 1452-1461 10 p.

Research output: Contribution to journalReview article

alpha 1-Antitrypsin Deficiency
Emphysema
Advisory Committees
Registries
Pulmonary Emphysema
2003
7 Citations (Scopus)

Active, specific immunotherapy for lung cancer: Hurdles and strategies using genetic modification

Korst, R. J. & Crystal, R., 1 Oct 2003, In : Annals of Thoracic Surgery. 76, 4, p. 1319-1326 8 p.

Research output: Contribution to journalReview article

Active Immunotherapy
Lung Neoplasms
Neoplasms
Immunotherapy
Immunity
2002
46 Citations (Scopus)

Systemic interleukin-6 responses following administration of adenovirus gene transfer vectors to humans by different routes

Harvey, B. G., McKinney, R. L., Rosengart, T., Lesser, M. L. & Crystal, R., 1 Jul 2002, In : Molecular Therapy. 6, 2, p. 287-297 11 p.

Research output: Contribution to journalReview article

Adenoviridae
Interleukin-6
Genes
Peripheral Vascular Diseases
Cystic Fibrosis
2001
22 Citations (Scopus)

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

Sondhi, D., Hackett, N. R., Apblett, R. L., Kaminsky, S. M., Pergolizzi, R. G. & Crystal, R., 20 Nov 2001, In : Archives of Neurology. 58, 11, p. 1793-1798 6 p.

Research output: Contribution to journalReview article

Neuronal Ceroid-Lipofuscinoses
Genetic Therapy
Lipofuscin
Central Nervous System Diseases
Neurodegenerative Diseases
2000

Myocardial revascularization: TMR and angiogenic therapy

Rosengart, T. K., Rahman, G. F. & Crystal, R., 1 Dec 2000, In : Cardiovascular Reviews and Reports. 21, 8, p. 414-420 7 p.

Research output: Contribution to journalReview article

Myocardial Revascularization
Intercellular Signaling Peptides and Proteins
Myocardium
Transmyocardial Laser Revascularization
Wounds and Injuries
1999
46 Citations (Scopus)

Therapeutic angiogenesis: Protein and gene therapy delivery strategies

Rosengart, T. K., Patel, S. R. & Crystal, R., 1 Jan 1999, In : European Journal of Preventive Cardiology. 6, 1, p. 29-40 12 p.

Research output: Contribution to journalReview article

Genetic Therapy
Proteins
Blood Vessels
Therapeutics
Angioplasty
1998
165 Citations (Scopus)

Gene therapy strategies for tumor antiangiogenesis

Kong, H. L. & Crystal, R., 18 Feb 1998, In : Journal of the National Cancer Institute. 90, 4, p. 273-286 14 p.

Research output: Contribution to journalReview article

Genetic Therapy
Neoplasms
Tourniquets
Neoplasm Micrometastasis
Growth
15 Citations (Scopus)

Pulmonary cell biology

Mason, R. J. & Crystal, R., 19 May 1998, In : American Journal of Respiratory and Critical Care Medicine. 157, 4 II SUPPL.

Research output: Contribution to journalReview article

Pulmonary Alveoli
Pulmonary Surfactants
Genetic Therapy
Lung Diseases
Cell Biology
16 Citations (Scopus)

Results of retroviral and adenoviral approaches to cancer gene therapy.

Yin, L. H., Fu, S. Q., Nanakorn, T., Garcia-Sanchez, F., Chung, I., Cote, R., Pizzorno, G., Hanania, E., Heimfeld, S., Crystal, R. & Deisseroth, A., 1 Dec 1998, In : Stem cells (Dayton, Ohio). 16 Suppl 1, p. 247-250 4 p.

Research output: Contribution to journalReview article

Neoplasm Genes
Genetic Therapy
Neoplasms
Cytosine Deaminase
Prodrugs
1995
54 Citations (Scopus)

A phase 1 study, in cystic fibrosis patients, of the safety, toxicity, and biological efficacy of a single administration of a replication deficient, recombinant adenovirus carrying the cDNA of the normal cystic fibrosis transmembrane conductance regulator gene in the lung

Crystal, R., Jaffe, A., Brody, S., Mastrangeli, A., McElvaney, N. G., Rosenfeld, M., Chu, C. S., Danel, C., Hay, J. & Eissa, T., 1 Jan 1995, In : Human Gene Therapy. 6, 5, p. 643-666 24 p.

Research output: Contribution to journalReview article

Gene Transfer Techniques
Cystic Fibrosis Transmembrane Conductance Regulator
Genetic Vectors
Clinical Trials, Phase I
Nasal Mucosa
1994
5 Citations (Scopus)
Gene Transfer Techniques
Cystic Fibrosis Transmembrane Conductance Regulator
Genetic Vectors
Cystic Fibrosis
Genetic Therapy
1993
15 Citations (Scopus)

Gene therapy for pulmonary diseases.

Rosenfeld, M. A., Ronald, G. & Crystal, R., 1 Oct 1993, In : Pathologie Biologie. 41, 8, p. 677-680 4 p.

Research output: Contribution to journalReview article

Gene therapy
Pulmonary diseases
Genetic Therapy
Lung Diseases
Genes
1992
2 Citations (Scopus)

α1-Antitrypsin augmentation therapy for chronic obstructive lung disorders

Crystal, R., 1 Jan 1992, In : Drugs of the Future. 17, 5, p. 387-393 7 p.

Research output: Contribution to journalReview article

Lung
Therapeutics
1990
344 Citations (Scopus)

α1-Antitrypsin deficiency, emphysema, and liver disease: Genetic basis and strategies for therapy

Crystal, R., 1 May 1990, In : Journal of Clinical Investigation. 85, 5, p. 1343-1352 10 p.

Research output: Contribution to journalReview article

Deficiency Diseases
alpha 1-Antitrypsin
Emphysema
Multigene Family
Codon
1989
92 Citations (Scopus)

Guidelines for the approach to the patient with severe hereditary alpha-1-antitrypsin deficiency

Buist, A. S., Burrows, B., Cohen, A., Crystal, R., Fallat, R. J., Gadek, J. E. & Turino, G. M., 1 Jan 1989, In : American Review of Respiratory Disease. 140, 5, p. 1494-1496 3 p.

Research output: Contribution to journalReview article

alpha 1-Antitrypsin Deficiency
Exercise Therapy
alpha 1-Antitrypsin
Irritants
Bronchodilator Agents
154 Citations (Scopus)

The alpha1-antitrypsin gene and its mutations. Clinical consequences and strategies for therapy

Crystal, R., Brantly, M. L., Hubbard, R. C., Curiel, D. T., States, D. J. & Holmes, M. D., 1 Jan 1989, In : Chest. 95, 1, p. 196-208 13 p.

Research output: Contribution to journalReview article

Mutation
Genes
Therapeutics
1985
106 Citations (Scopus)

Oxidants, antioxidants and the pathogenesis of emphysema.

Cantin, A. & Crystal, R., 1 Jan 1985, In : European journal of respiratory diseases. Supplement. 139, p. 7-17 11 p.

Research output: Contribution to journalReview article

Emphysema
Oxidants
Antioxidants
Respiratory System
Connective Tissue
1984
17 Citations (Scopus)

Current concepts of the pathogenesis of fibrosis: lessons from pulmonary fibrosis.

Rennard, S. I., Bitterman, P. B. & Crystal, R., 1 Jan 1984, In : Progress in Clinical and Biological Research. 154, p. 359-377 19 p.

Research output: Contribution to journalReview article

Pulmonary Alveoli
Primary Myelofibrosis
Pulmonary Fibrosis
Fibronectins
Cell Division
404 Citations (Scopus)

Interstitial Lung Diseases of Unknown Cause: Disorders Characterized by Chronic Inflammation of the Lower Respiratory Tract

Crystal, R., Bitterman, P. B., Rennard, S. I., Hance, A. J. & Keogh, B. A., 19 Jan 1984, In : New England Journal of Medicine. 310, 3, p. 154-166 13 p.

Research output: Contribution to journalReview article

Interstitial Lung Diseases
Respiratory System
Inflammation
210 Citations (Scopus)

Interstitial Lung Diseases of Unknown Cause: Disorders Characterized by Chronic Inflammation of the Lower Respiratory Tract

Crystal, R., Bitterman, P. B., Rennard, S. I., Hance, A. J. & Keogh, B. A., 26 Jan 1984, In : New England Journal of Medicine. 310, 4, p. 235-244 10 p.

Research output: Contribution to journalReview article

Interstitial Lung Diseases
Sarcoidosis
Respiratory System
Inflammation
Pulmonary Sarcoidosis
93 Citations (Scopus)

Pathogenesis of the granulomatous lung diseases

Hunninghake, G. W., Garrett, K. C., Richerson, H. B., Fantone, J. C., Ward, P. A., Rennard, S. I., Bitterman, P. B. & Crystal, R., 1 Nov 1984, In : American Review of Respiratory Disease. 130, 3, p. 476-496 21 p.

Research output: Contribution to journalReview article

Granuloma
Lung Diseases
Macrophages
T-Lymphocytes
Lung
35 Citations (Scopus)

Role of connective tissue proteases in the pathogenesis of chronic inflammatory lung disease.

Gadek, J. E., Fells, G. A., Zimmerman, R. L. & Crystal, R., 1 Apr 1984, In : Environmental Health Perspectives. 55, p. 297-306 10 p.

Research output: Contribution to journalReview article

Pulmonary diseases
Connective Tissue
Lung Diseases
Peptide Hydrolases
Tissue
1983
23 Citations (Scopus)

Response of the lower respiratory tract to injury. Mechanisms of repair of the parenchymal cells of the alveolar wall

Rennard, S. I., Bitterman, P. B. & Crystal, R., 1 Jan 1983, In : Chest. 84, 6, p. 735-739 5 p.

Research output: Contribution to journalReview article

Respiratory System
Cell Wall
Fibrosis
Wounds and Injuries
Endothelial Cells