Results 61 to 70 of about 1,182,904 (110)
Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders. [PDF]
J Blood MedWinkler R +5 more
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Rules, risks, and reality: rethinking gene therapy eligibility. [PDF]
Blood AdvKarsenty CL, Ngwube AI.
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Therapeutic frontiers in ALS: iPSC-based drug discovery, cell therapy, and gene therapy-Advances through 2026. [PDF]
Regen TherMorimoto S +3 more
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Gene Therapy Advancements in Age-Related Macular Degeneration Treatment. [PDF]
CellsAmaxilati E +6 more
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Experimental Platform for Screening and Validation of BacNa<sub>v</sub> Gene Therapy Candidates. [PDF]
Circ Arrhythm ElectrophysiolWu T, Nguyen HX, Siu YY, Li Y, Bursac N.
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Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models. [PDF]
Nat CommunCozmescu CA +21 more
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New England Journal of Medicine, 2019
Gene therapy medicinal products (GTMPs) are one of the most promising biopharmaceuticals, which are beginning to show encouraging results. The broad clinical research activity has been addressed mainly to cancer, primarily to those cancers that do not respond well to conventional treatment.
Ana, Del Pozo-Rodríguez +6 more
openaire +4 more sources
Gene therapy medicinal products (GTMPs) are one of the most promising biopharmaceuticals, which are beginning to show encouraging results. The broad clinical research activity has been addressed mainly to cancer, primarily to those cancers that do not respond well to conventional treatment.
Ana, Del Pozo-Rodríguez +6 more
openaire +4 more sources
2022
Gene therapy drugs and products are non-classic bioproducts designed and established for precision and/or personalized medicine. Gene therapy drugs are developed primarily based on the non-viral or modified-viral tools to restore the function of the missing or defective genes in the target cells.
Shahryari, A. +7 more
openaire +2 more sources
Gene therapy drugs and products are non-classic bioproducts designed and established for precision and/or personalized medicine. Gene therapy drugs are developed primarily based on the non-viral or modified-viral tools to restore the function of the missing or defective genes in the target cells.
Shahryari, A. +7 more
openaire +2 more sources
Fetal Diagnosis and Therapy, 1987
Severe genetic disorders are potentially correctable by the addition of a normal gene into tissues. Although the technical problems involving integration, stable expression, and insertional damage to the treated cell are not yet fully solved, enough scientific progress has already been made to consider somatic cell gene therapy acceptable from both the
A, Drugan, O J, Miller, M I, Evans
openaire +2 more sources
Severe genetic disorders are potentially correctable by the addition of a normal gene into tissues. Although the technical problems involving integration, stable expression, and insertional damage to the treated cell are not yet fully solved, enough scientific progress has already been made to consider somatic cell gene therapy acceptable from both the
A, Drugan, O J, Miller, M I, Evans
openaire +2 more sources