Human Gene Therapy definition

Human Gene Therapy means the in vivo administration of genetic material directly into a human being using viral vectors, including but not limited to AAV Vectors, to transfer such material into the patient’s own cells for the purpose of producing proteins or other macromolecules that are expressed in or secreted from the transduced cells for therapeutic or prophylactic purposes, where (i) the expression of such proteins or other macromolecules is regulated through the administration of [***] or another [***] Dimerizer, or (ii) the inserted genetic material is not regulated through the administration of a Dimerizer. For clarity, “Human Gene Therapy” shall not include (a) any use where genetic material is inserted into a cell ex vivo, including without limitation any use for a cancer or non-cancer vaccine or immunotherapeutic product or (b) any use where activation of genetic material for any function other than expression is regulated through the administration of Dimerizer.
Sample 1Sample 2
Human Gene Therapy means the in vivo administration of genetic material directly into a human being using viral vectors, including but not limited to AAV Vectors, to transfer such material into the patient’s own cells for the purpose of producing proteins or other macromolecules that are expressed in or secreted from the transduced cells for therapeutic or prophylactic purposes, where (i) the expression of such proteins or other macromolecules is regulated through the administration of [...***...] or another [...***...] Dimerizer, or (ii) the inserted genetic material is not regulated through the administration of a Dimerizer. For clarity, “Human Gene Therapy” shall not include (a) any use where genetic material is inserted into a cell ex vivo, including without limitation any use for a cancer or non-cancer vaccine or immunotherapeutic product or (b) any use where activation of genetic material for any function other than expression is regulated through the administration of Dimerizer.
Sample 1Sample 2
Human Gene Therapy shall not include (a) any use where genetic material is inserted into a cell ex vivo, including without limitation any use for a cancer or non-cancer vaccine or immunotherapeutic product or (b) any use where activation of genetic material for any function other than expression is regulated through the administration of Dimerizer.
Sample 1

Examples of Human Gene Therapy in a sentence

  • Human Gene Therapy Cellular therapies for muscular dystrophies: frustrations and clinical successes (doi: 10.1089/hum.2015.139) This article has been peer-reviewed and accepted for publication, but has yet to undergo copyediting and proof correction.

  • Janich, B.J. Roessler and G.L. Amidon, Role of integrin expression in adenovirus-mediated gene delivery to the intestinal epithelium, Human Gene Therapy, 9, 561 (1998).

  • To whom requests for reprints should be addressed, at Division of Human Gene Therapy, Departments of Medicine, Pathology, and Sur- gery, and The Gene Therapy Center, University of Alabama at Birming- ham, Birmingham, Alabama 35233.

  • They are becoming the reagents of choice for efficient DNA and siRNA transfections as demonstrated by their increasing use in numerous studies published in high impact factor journals including Journal of Biological Chemistry, Nucleic Acids Research, PLOS ONE, PLOS Pathogen, Human Gene Therapy and more.

  • Guidance for Industry: Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs) (U.S. Department of Health and Human Services).

  • Testing of Retroviral Vector-Based Human Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-up; Guidance for Industry, January 2020.

  • Testing of Retroviral Vector- Based Human Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Guidance for Industry” is available at this website: https://www.fda.gov/media/113790/download.

  • This guidance applies to human gene therapy products and to combination products1 that contain a human gene therapy in combination with a drug or device.2 This guidance finalizes the draft guidance of the same title dated July 2018 and supersedes the document entitled “Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs),” dated April 2008 (April 2008 guidance).

  • Gene trans- fer studies were performed in part at the University of Alabama at Birmingham, Corroborative Laboratories for Human Gene Therapy Trials.

  • They are becoming the reagents of choice for efficient DNA and siRNA transfections as demonstrated by their increasing use in numerous studies published in high impact factor journals including Journal of Biological Chemistry, Nucleic Acids Research, PLOS ONE, PLOS Pathogen, Human Gene Therapy, Cell reports, EMBO Jand more.

Related to Human Gene Therapy

  • Gene Therapy means a product or service for the treatment or prevention of a disease that utilizes ex vivo or in vivo delivery (via viral or nonviral gene transfer methods or systems) of Genetic Material, including any cell incorporating Genetic Material.

  • Step therapy means a protocol or

  • in vitro diagnostic medical device means any medical device which is a reagent, reagent product, calibrator, control material, kit, instrument, apparatus, equipment, software or system, whether used alone or in combination, intended by the manufacturer to be used in vitro for the examination of specimens, including blood and tissue donations, derived from the human body, solely or principally for the purpose of providing information:

  • Medical cannabis means the same as that term is defined in Section 26-61a-102.

  • Collaborative drug therapy management means participation by an authorized pharmacist and a physician in the management of drug therapy pursuant to a written community practice protocol or a written hospital practice protocol.

  • Therapeutic school means a residential group living facility:

  • Diagnosis means the definition of the nature of the Client's disorder. When formulating the Diagnosis of Client, CONTRACTOR shall use the diagnostic codes and axes as specified in the most current edition of the DSM published by the American Psychiatric Association. DSM diagnoses will be recorded on all IRIS documents, as appropriate.

  • Vaccine means a specially prepared antigen which, upon administration to a person, will result in immunity and, specifically for the purposes of this rule, shall mean influenza and pneumococcal vaccines.

  • Biological diversity means the variability among living organisms from all sources including, inter alia, terrestrial, marine and other aquatic ecosystems and the ecological complexes of which they are part; this includes diversity within species, between species and of ecosystems.

  • Commercialization or “Commercialize” means activities directed to marketing, promoting, research and development as required, manufacturing for sale, offering for sale, distributing, importing or selling a product, including sub-licensing or sub-contracting of these activities.

  • Clinical laboratory means a facility for the microbiological, serological, chemical, hematological, radiobioassay, cytological, immunohematological, pathological, or other examination of materials derived from the human body for the purpose of providing information for the diagnosis, prevention, or treatment of a disease or assessment of a medical condition.

  • Diagnostic Product means any test or assay for diagnosing or detecting a disease, disorder, medical condition, or symptom.

  • Medical cannabis card means the same as that term is defined in Section 26-61a-102.

  • Development Candidate means a Collaboration Compound designated by Biogen Idec as a Development Candidate in accordance with Section 2.6.

  • Opioid antagonist means a drug that binds to opioid

  • Moving beam radiation therapy means radiation therapy with any planned displacement of radiation field or patient relative to each other, or with any planned change of absorbed dose distribution. It includes arc, skip, conformal, intensity modulation and rotational therapy.

  • Biomarker means a parameter or characteristic in a patient or Patient Sample, the measurement of which is useful (a) for purposes of selecting appropriate therapies or patient populations or monitoring disease susceptibility, severity or state, or monitoring therapies for such patient and/or (b) for predicting the outcome of a particular treatment of such patient.

  • Clinical means having a significant relationship, whether real or potential, direct or indirect, to the actual rendering or outcome of dental care, the practice of dentistry, or the quality of dental care being rendered to a patient;

  • scientific research means any activity in the field of natural or applied science for the extension of knowledge;

  • Step therapy protocol means a protocol or program that establishes the specific

  • medical food means a food that is intended for the dietary treatment of a disease or condition for which nutritional requirements are established by medical evaluation and is formulated to be consumed or administered enterally under the direction of a Practitioner.

  • Licensed mental health professional or "LMHP" means a physician, licensed clinical psychologist, licensed professional counselor, licensed clinical social worker, licensed substance abuse treatment practitioner, licensed marriage and family therapist, certified psychiatric clinical nurse specialist, licensed behavior analyst, or licensed psychiatric/mental health nurse practitioner.

  • Lead Compound means any compound of lead other than galena which, when treated in the manner described below, yields to an aqueous solution of hydrochloric acid a quantity soluble lead compound exceeding, when calculated as lead monoxide, five percent of the dry weight of the portion taken analysis.

  • Opioid antidote means any drug, regardless of dosage amount or method of administration, which has been approved by the United States Food and Drug Administration (FDA) for the treatment of an opioid overdose. “Opioid antidote” includes, but is not limited to, naloxone hydrochloride, in any dosage amount, which is administered through nasal spray or any other FDA-approved means or methods.

  • Collaboration Compound means any of the following: (a) FG-4592, (b) any HIF Compound (other than FG-4592) that is added to this Agreement pursuant to Section 3.6, and (c) any salts, esters, complexes, chelates, crystalline and amorphous morphic forms, pegylated forms, enantiomers (excluding regioisomers), prodrugs, solvates, metabolites and catabolites of any of the foregoing ((a) or (b)).

  • Clinical nurse specialist means a registered nurse with relevant post-basic qualifications and 12 months’ experience working in the clinical area of his/her specified post-basic qualification, or a minimum of four years’ post-basic registration experience, including three years’ experience in the relevant specialist field and who satisfies the local criteria.