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1. Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.

Author

O'Reilly, Marina, Federoff, Howard J, Fong, Yuman, Kohn, Donald B, Patterson, Amy P, Ahmed, Nabil, Asokan, Aravind, Boye, Shannon E, Crystal, Ronald G, De Oliveira, Satiro, Gargiulo, Linda, Harper, Scott Q, Ikeda, Yasuhiro, Jambou, Robert, Montgomery, Maureen, Prograis, Lawrence, Rosenthal, Eugene, Sterman, Daniel H, Vandenberghe, Luk H, Zoloth, Laurie, Abedi, Mehrdad, Adair, Jennifer, Adusumilli, Prasad S, Goins, William F, Gray, Jhanelle, Monahan, Paul, Popplewell, Leslie, Sena-Esteves, Miguel, Tannous, Bakhos, Weber, Thomas, Wierda, William, Gopal-Srivastava, Rashmi, McDonald, Cheryl L, Rosenblum, Daniel, and Corrigan-Curay, Jacqueline

Subjects
Animals, Education, Continuing, Genetic Research, Genetic Therapy: economics, ethics, legislation & jurisprudence, Genetic Vectors, Humans, National Institutes of Health (U.S.), Stem Cell Research, Stem Cell Transplantation, Transduction, Genetic, United States
Abstract

Recently, the gene therapy field has begun to experience clinical successes in a number of different diseases using various approaches and vectors. The workshop Gene Therapy: Charting a Future Course, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities, brought together early and mid-career researchers to discuss the key scientific challenges and opportunities, ethical and communication issues, and NIH and foundation resources available to facilitate further clinical advances.

Published
2014

5. Genome Editing Technologies: Defining a Path to Clinic

Author

Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences, Zhang, Feng, Corrigan-Curay, Jacqueline, O'Reilly, Marina, Kohn, Donald B, Cannon, Paula M, Bao, Gang, Bushman, Frederic D, Carroll, Dana, Cathomen, Toni, Joung, J Keith, Roth, David, Sadelain, Michel, Scharenberg, Andrew M, von Kalle, Christof, Jambou, Robert, Rosenthal, Eugene, Hassani, Morad, Singh, Aparna, Porteus, Matthew H, Massachusetts Institute of Technology. Department of Brain and Cognitive Sciences, Zhang, Feng, Corrigan-Curay, Jacqueline, O'Reilly, Marina, Kohn, Donald B, Cannon, Paula M, Bao, Gang, Bushman, Frederic D, Carroll, Dana, Cathomen, Toni, Joung, J Keith, Roth, David, Sadelain, Michel, Scharenberg, Andrew M, von Kalle, Christof, Jambou, Robert, Rosenthal, Eugene, Hassani, Morad, Singh, Aparna, and Porteus, Matthew H

Published
2017

6. Genome Editing Technologies: Defining a Path to Clinic: Genomic Editing: Establishing Preclinical Toxicology Standards, Bethesda, Maryland 10 June 2014

Author

Corrigan-Curay, Jacqueline, O'Reilly, Marina, Kohn, Donald B, Cannon, Paula M, Bao, Gang, Bushman, Frederic D, Carroll, Dana, Cathomen, Toni, Joung, J Keith, Roth, David, Sadelain, Michel, Scharenberg, Andrew M, von Kalle, Christof, Zhang, Feng, Jambou, Robert, Rosenthal, Eugene, Hassani, Morad, Singh, Aparna, and Porteus, Matthew H

Subjects
Meeting Report
Published
2015

7. T-Cell Immunotherapy: Looking Forward: T Cell Immunotherapy: Optimizing Trial Design Bethesda, Maryland 10-11 September 2013

Author

Corrigan-Curay, Jacqueline, Kiem, Hans-Peter, Baltimore, David, O'Reilly, Marina, Brentjens, Renier J, Cooper, Laurence, Forman, Stephen, Gottschalk, Stephen, Greenberg, Philip, Junghans, Richard, Heslop, Helen, Jensen, Michael, Mackall, Crystal, June, Carl, Press, Oliver, Powell, Daniel, Ribas, Antoni, Rosenberg, Steven, Sadelain, Michel, Till, Brian, Patterson, Amy P, Jambou, Robert C, Rosenthal, Eugene, Gargiulo, Linda, Montgomery, Maureen, and Kohn, Donald B

Subjects
Meeting Report
Published
2014

8. Gene Therapy: Charting a Future Course—Summary of a National Institutes of Health Workshop, April 12, 2013

Author

Kohn, Donald B., Federoff, Howard J., Vandenberghe, Luk H., Asokan, Aravind, Abedi, Mehrdad, Zoloth, Laurie, Adusumilli, Prasad S., Prograis, Lawrence, O'Reilly, Marina, Jambou, Robert, Boye, Shannon E., Monahan, Paul, Gopal-Srivastava, Rashmi, Gargiulo, Linda, Gray, Jhanelle, Sena-Esteves, Miguel, Ikeda, Yasuhiro, Rosenthal, Eugene, Wierda, William, Harper, Scott Q., Crystal, Ronald G., Ahmed, Nabil, Montgomery, Maureen, Weber, Thomas, Popplewell, Leslie, Patterson, Amy P., De Oliveira, Satiro, Adair, Jennifer, Goins, William F., Sterman, Daniel H., Tannous, Bakhos, and Fong, Yuman

Subjects
education
Abstract

Recently, the gene therapy field has begun to experience clinical successes in a number of different diseases using various approaches and vectors. The workshop Gene Therapy: Charting a Future Course, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities, brought together early and mid-career researchers to discuss the key scientific challenges and opportunities, ethical and communication issues, and NIH and foundation resources available to facilitate further clinical advances.

Published
2014
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11. Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy: Retroviral and Lentiviral Vectors for Long-Term Gene Correction: Clinical Challenges in Vector and Trial Design Bethesda, Maryland, USA 9–10 December 2010

Author

Corrigan-Curay, Jacqueline, Cohen-Haguenauer, Odile, O'Reilly, Marina, Ross, Susan R., Fan, Hung, Rosenberg, Naomi, Somia, Nikunj, King, Nancy, Friedmann, Theodore, Dunbar, Cynthia, Aiuti, Alessandro, Naldini, Luigi, Baum, Christopher, von Kalle, Christof, Kiem, Hans-Peter, Montini, Eugenio, Bushman, Frederic, Sorrentino, Brian P, Carrondo, Manuel, Malech, Harry, Gahrton, Gösta, Shapiro, Robyn, Wolff, Linda, Rosenthal, Eugene, Jambou, Robert, Zaia, John, and Kohn, Donald B

Subjects
Meeting Report
Published
2012

12. Genome Editing Technologies: Defining a Path to Clinic

Author

Corrigan-Curay, Jacqueline, primary, O'Reilly, Marina, additional, Kohn, Donald B, additional, Cannon, Paula M, additional, Bao, Gang, additional, Bushman, Frederic D, additional, Carroll, Dana, additional, Cathomen, Toni, additional, Joung, J Keith, additional, Roth, David, additional, Sadelain, Michel, additional, Scharenberg, Andrew M, additional, von Kalle, Christof, additional, Zhang, Feng, additional, Jambou, Robert, additional, Rosenthal, Eugene, additional, Hassani, Morad, additional, Singh, Aparna, additional, and Porteus, Matthew H, additional

Published
2015
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13. T-Cell Immunotherapy: Looking Forward

Author

Corrigan-Curay, Jacqueline, primary, Kiem, Hans-Peter, additional, Baltimore, David, additional, O'Reilly, Marina, additional, Brentjens, Renier J, additional, Cooper, Laurence, additional, Forman, Stephen, additional, Gottschalk, Stephen, additional, Greenberg, Philip, additional, Junghans, Richard, additional, Heslop, Helen, additional, Jensen, Michael, additional, Mackall, Crystal, additional, June, Carl, additional, Press, Oliver, additional, Powell, Daniel, additional, Ribas, Antoni, additional, Rosenberg, Steven, additional, Sadelain, Michel, additional, Till, Brian, additional, Patterson, Amy P, additional, Jambou, Robert C, additional, Rosenthal, Eugene, additional, Gargiulo, Linda, additional, Montgomery, Maureen, additional, and Kohn, Donald B, additional

Published
2014
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15. Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012

Author

O'Reilly, Marina, primary, Kohn, Donald B., additional, Bartlett, Jeffrey, additional, Benson, Janet, additional, Brooks, Philip J., additional, Byrne, Barry J., additional, Camozzi, Carlos, additional, Cornetta, Kenneth, additional, Crystal, Ronald G., additional, Fong, Yuman, additional, Gargiulo, Linda, additional, Gopal-Srivastava, Rashmi, additional, High, Katherine A., additional, Jacobson, Samuel G., additional, Jambou, Robert C., additional, Montgomery, Maureen, additional, Rosenthal, Eugene, additional, Samulski, R. Jude, additional, Skarlatos, Sonia I., additional, Sorrentino, Brian, additional, Wilson, James M., additional, Xie, Yun, additional, and Corrigan-Curay, Jacqueline, additional

Published
2013
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16. Challenges in Vector and Trial Design Using Retroviral Vectors for Long-Term Gene Correction in Hematopoietic Stem Cell Gene Therapy

Author

Corrigan-Curay, Jacqueline, primary, Cohen-Haguenauer, Odile, additional, O'Reilly, Marina, additional, Ross, Susan R., additional, Fan, Hung, additional, Rosenberg, Naomi, additional, Somia, Nikunj, additional, King, Nancy, additional, Friedmann, Theodore, additional, Dunbar, Cynthia, additional, Aiuti, Alessandro, additional, Naldini, Luigi, additional, Baum, Christopher, additional, von Kalle, Christof, additional, Kiem, Hans-Peter, additional, Montini, Eugenio, additional, Bushman, Frederic, additional, Sorrentino, Brian P, additional, Carrondo, Manuel, additional, Malech, Harry, additional, Gahrton, Gösta, additional, Shapiro, Robyn, additional, Wolff, Linda, additional, Rosenthal, Eugene, additional, Jambou, Robert, additional, Zaia, John, additional, and Kohn, Donald B, additional

Published
2012
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21. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium

Author

Rosenfeld, Melissa A., primary, Yoshimura, Kunihiko, additional, Trapnell, Bruce C., additional, Yoneyama, Koichi, additional, Rosenthal, Eugene R., additional, Dalemans, Wilfried, additional, Fukayama, Masashi, additional, Bargon, Joachim, additional, Stier, Larue E., additional, Stratford-Perricaudet, Leslie, additional, Perricaudet, Michel, additional, Guggino, William B., additional, Pavirani, Andrea, additional, Lecocq, Jean-Pierre, additional, and Crystal, Ronald G., additional

Published
1992
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39. Curry Café.

Author

Rosenthal, Eugene

Abstract

Features the Curry Café in Islip, New York. Interior decoration of the restaurant; Food specialty; Menu offered.

Published
2003

40. The National Institutes of Health Oversight of Human Gene Transfer Research: Enhancing Science and Safety.

Author

O'Reilly M, Jambou R, Rosenthal E, Montgomery M, Hassani M, Gargiulo L, and Corrigan-Curay J

Subjects
Animals, Clinical Trials as Topic, Genetic Therapy ethics, Humans, Informed Consent ethics, Informed Consent legislation & jurisprudence, National Institutes of Health (U.S.), Patient Safety legislation & jurisprudence, Practice Guidelines as Topic, Research Design, Translational Research, Biomedical ethics, United States, DNA, Recombinant therapeutic use, Drug and Narcotic Control legislation & jurisprudence, Genetic Therapy legislation & jurisprudence, Genetic Vectors therapeutic use, Translational Research, Biomedical legislation & jurisprudence
Abstract

The National Institutes of Health (NIH) oversight of human gene transfer research, which is defined as the deliberate transfer of recombinant and/or synthetic nucleic acid molecules to humans, originates with the NIH Guidelines for Research Involving Recombinant or Synthetic Nucleic Acid Molecules (NIH Guidelines). The NIH Guidelines, which were first published in the Federal Register almost 40 years ago, have been amended numerous times to remain responsive to scientific progress and to clearly define the responsibilities of NIH, the Recombinant DNA Advisory Committee (RAC), investigators, and institutions. Human gene transfer trials conducted at clinical sites in the United States (USA) are subject to the NIH Guidelines if they are conducted at, or sponsored by, an institution that receives any support for recombinant or synthetic nucleic acid research from the NIH. Human gene transfer trials conducted either in the USA or abroad are also subject to the NIH Guidelines if the investigational agent was developed with NIH funds and the institution that developed the investigational materials sponsors or participates in these projects. Trials are registered with the NIH Office Biotechnology Activities (OBA) and there are ongoing reporting requirements. Each new trial is reviewed by the RAC, and those that are novel or raise unique ethical or social issues are selected for review at quarterly public RAC meetings. The RAC also advises the NIH on policy and other matters relating to clinical gene transfer research and biosafety.

Published
2015
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41. NIH oversight of human gene transfer research involving retroviral, lentiviral, and adeno-associated virus vectors and the role of the NIH recombinant DNA advisory committee.

Author

O'Reilly M, Shipp A, Rosenthal E, Jambou R, Shih T, Montgomery M, Gargiulo L, Patterson A, and Corrigan-Curay J

Subjects
Animals, Clinical Trials as Topic, Dependovirus genetics, Gene Transfer Techniques, Genetic Therapy methods, Genetic Vectors, Humans, Practice Guidelines as Topic, Retroviridae genetics, United States, Advisory Committees statistics & numerical data, DNA, Recombinant genetics, Genetic Therapy standards, National Institutes of Health (U.S.)
Abstract

In response to public and scientific concerns regarding human gene transfer research, the National Institutes of Health (NIH) developed a transparent oversight system that extends to human gene transfer protocols that are either conducted with NIH funding or conducted at institutions that receive NIH funding for recombinant DNA research. The NIH Recombinant DNA Advisory Committee (RAC) has been the primary advisory body to NIH regarding the conduct of this research. Human gene transfer research proposals that are subject to the NIH Guidelines for Research Involving Recombinant DNA Molecules (NIH Guidelines) must be submitted to the NIH Office of Biotechnology Activities (OBA), and protocols that raise novel scientific, safety, medical, ethical, or social issues are publicly discussed at the RAC's quarterly public meetings. OBA also convenes gene transfer safety symposia and policy conferences to provide a public forum for scientific experts to discuss emerging issues in the field. This transparent system of review promotes the rapid exchange of important scientific information and dissemination of data. The goal is to optimize the conduct of individual research protocols and to advance gene transfer research generally. This process has fostered the development of retroviral, lentiviral, and adeno-associated viral vector mediated gene delivery., (Copyright © 2012 Elsevier Inc. All rights reserved.)

Published
2012
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