FRINK Linked Data Fragments server

Matches in SemOpenAlex for { <https://semopenalex.org/work/W4387563224> ?p ?o ?g. }

• W4387563224 abstract "Human Gene TherapyAhead of Print EditorialFree AccessSpecial Editorial Feature: Learning from Our FailuresTerence R. FlotteTerence R. FlotteEditor-in-ChiefSearch for more papers by this authorPublished Online:12 Oct 2023https://doi.org/10.1089/hum.2023.29246.editorialAboutSectionsPDF/EPUB Permissions & CitationsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail In this issue of Human Gene Therapy, we provide a venue for a deeper discussion of the tragic case of Mr. Terry Horgan, a 27-year old man with Duchenne muscular dystrophy (DMD) caused by a mutation in exon 1 of the Dystrophin gene. Terry underwent treatment with a high-dose (1 × 1014 vg/kg) intravenous infusion of recombinant adeno-associated virus 9 (rAAV9) vector delivering a CRISPR-based transactivator, making him the first person to receive a CRISPR-based treatment for the disease. Unfortunately, Terry suffered severe cardiopulmonary decline 6 days after receiving treatment, which led to his death. The scientific and clinical details of the case are described elsewhere.1 We, the senior editorial team of Human Gene Therapy, have chosen to provide the venue of this special editorial feature, not as a forum to discuss the science, but rather as a channel for those involved in this case to share additional unique perspectives from families and providers involved in cutting-edge human trials that do NOT go well.Throughout the history of human subjects research, there have been numerous tragic failures that have paved the way for later successes. This includes decades of failures in trials of chemotherapy for pediatric leukemias, bone marrow and solid organ transplants, and cancer surgeries.2 In each of these cases, intrepid patients and their providers chose to study their failures and their partial successes and to learn from them, and gene therapy researchers must follow that example. We must resist the temptation to only trumpet “successful” trial results. Of course, we always strive for positive outcomes in each individual pateint, but any experimental result that increases our knowledge is a success for the field as a whole. That is why we MUST as a field embrace our failures as much or more than our successes. It is only by studying, learning, and sharing undesired outcomes that we can improve as a clinical discipline.References1. Lek A, Wong B, Keeler A, et al. Death after high-dose rAAV9 gene therapy in a patient with Duchenne's muscular dystrophy. N Engl J Med 2023;389(13):1203–1210; doi: 10.1056/NEJMoa2307798. Crossref, Medline, Google Scholar2. Mukherjee S. The Emperor of All Maladies: A Biography of Cancer. Simon & Schuster: London, United Kingdom; 2010. Google ScholarFiguresReferencesRelatedDetails Volume 0Issue 0 InformationCopyright 2023, by Mary Ann Liebert, Inc., publishersTo cite this article:Terence R. Flotte.Special Editorial Feature: Learning from Our Failures.Human Gene Therapy.ahead of printhttp://doi.org/10.1089/hum.2023.29246.editorialOnline Ahead of Print:October 12, 2023PDF download" @default.
• W4387563224 created "2023-10-13" @default.
• W4387563224 creator A5010005871 @default.
• W4387563224 date "2023-10-12" @default.
• W4387563224 modified "2023-10-18" @default.
• W4387563224 title "Special Editorial Feature: Learning from Our Failures" @default.
• W4387563224 cites W4387077861 @default.
• W4387563224 doi "https://doi.org/10.1089/hum.2023.29246.editorial" @default.
• W4387563224 hasPubMedId "https://pubmed.ncbi.nlm.nih.gov/37823803" @default.
• W4387563224 hasPublicationYear "2023" @default.
• W4387563224 type Work @default.
• W4387563224 citedByCount "0" @default.
• W4387563224 crossrefType "journal-article" @default.
• W4387563224 hasAuthorship W4387563224A5010005871 @default.
• W4387563224 hasConcept C104317684 @default.
• W4387563224 hasConcept C111599444 @default.
• W4387563224 hasConcept C126322002 @default.
• W4387563224 hasConcept C23161992 @default.
• W4387563224 hasConcept C2778943923 @default.
• W4387563224 hasConcept C52119013 @default.
• W4387563224 hasConcept C54355233 @default.
• W4387563224 hasConcept C554144382 @default.
• W4387563224 hasConcept C60644358 @default.
• W4387563224 hasConcept C71924100 @default.
• W4387563224 hasConcept C86803240 @default.
• W4387563224 hasConcept C95457728 @default.
• W4387563224 hasConcept C98108389 @default.
• W4387563224 hasConceptScore W4387563224C104317684 @default.
• W4387563224 hasConceptScore W4387563224C111599444 @default.
• W4387563224 hasConceptScore W4387563224C126322002 @default.
• W4387563224 hasConceptScore W4387563224C23161992 @default.
• W4387563224 hasConceptScore W4387563224C2778943923 @default.
• W4387563224 hasConceptScore W4387563224C52119013 @default.
• W4387563224 hasConceptScore W4387563224C54355233 @default.
• W4387563224 hasConceptScore W4387563224C554144382 @default.
• W4387563224 hasConceptScore W4387563224C60644358 @default.
• W4387563224 hasConceptScore W4387563224C71924100 @default.
• W4387563224 hasConceptScore W4387563224C86803240 @default.
• W4387563224 hasConceptScore W4387563224C95457728 @default.
• W4387563224 hasConceptScore W4387563224C98108389 @default.
• W4387563224 hasLocation W43875632241 @default.
• W4387563224 hasLocation W43875632242 @default.
• W4387563224 hasOpenAccess W4387563224 @default.
• W4387563224 hasPrimaryLocation W43875632241 @default.
• W4387563224 hasRelatedWork W2077589865 @default.
• W4387563224 hasRelatedWork W2081357017 @default.
• W4387563224 hasRelatedWork W2141619720 @default.
• W4387563224 hasRelatedWork W2186989880 @default.
• W4387563224 hasRelatedWork W2405656580 @default.
• W4387563224 hasRelatedWork W2642604616 @default.
• W4387563224 hasRelatedWork W3162049976 @default.
• W4387563224 hasRelatedWork W3163009052 @default.
• W4387563224 hasRelatedWork W4293199045 @default.
• W4387563224 hasRelatedWork W4381712632 @default.
• W4387563224 isParatext "false" @default.
• W4387563224 isRetracted "false" @default.
• W4387563224 workType "article" @default.