Ethical challenges for a new generation of early-phase pediatric gene therapy trials.

Autor: Iyer AA; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA., Saade D; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA., Bharucha-Goebel D; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA.; Children's National Hospital, Washington, DC, USA., Foley AR; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA., Averion G'; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA., Paredes E; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA., Gray S; University of Texas Southwestern Viral Vector Facility, Dallas, TX, USA., Bönnemann CG; Neuromuscular and Neurogenetic Disorders of Childhood Section, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD, USA., Grady C; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA., Hendriks S; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA., Rid A; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA. annette.rid@nih.gov.

Publikováno v: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2021 Nov; Vol. 23 (11), pp. 2057-2066. Date of Electronic Publication: 2021 Jul 07.

Cellular reprogramming with ATOH1, GFI1, and POU4F3 implicate epigenetic changes and cell-cell signaling as obstacles to hair cell regeneration in mature mammals.

Autor: Iyer AA; Department of Molecular & Human Genetics, Baylor College of Medicine, Houston, United States., Hosamani I; Department of Molecular & Human Genetics, Baylor College of Medicine, Houston, United States., Nguyen JD; Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine of the University of Southern California, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Biology at USC, Los Angeles, United States., Cai T; Program in Developmental Biology, Baylor College of Medicine, Houston, United States., Singh S; Department of Neuroscience, Baylor College of Medicine, Houston, United States., McGovern MM; Department of Neuroscience, Baylor College of Medicine, Houston, United States., Beyer L; Department of Otolaryngology-Head and Neck Surgery, University of Michigan, Ann Arbor, United States., Zhang H; Department of Neuroscience, Baylor College of Medicine, Houston, United States., Jen HI; Program in Developmental Biology, Baylor College of Medicine, Houston, United States.; Department of Neuroscience, Baylor College of Medicine, Houston, United States., Yousaf R; Department of Neuroscience, Baylor College of Medicine, Houston, United States., Birol O; Program in Developmental Biology, Baylor College of Medicine, Houston, United States., Sun JJ; Department of Neuroscience, Baylor College of Medicine, Houston, United States., Ray RS; Department of Neuroscience, Baylor College of Medicine, Houston, United States., Raphael Y; Department of Otolaryngology-Head and Neck Surgery, University of Michigan, Ann Arbor, United States., Segil N; Department of Stem Cell Biology and Regenerative Medicine, Keck School of Medicine of the University of Southern California, Eli and Edythe Broad Center for Regenerative Medicine and Stem Cell Biology at USC, Los Angeles, United States.; Caruso Department of Otolaryngology-Head and Neck Surgery, Keck School of Medicine of the University of Southern California, Los Angeles, United States., Groves AK; Department of Molecular & Human Genetics, Baylor College of Medicine, Houston, United States.; Program in Developmental Biology, Baylor College of Medicine, Houston, United States.; Department of Neuroscience, Baylor College of Medicine, Houston, United States.

Publikováno v: ELife [Elife] 2022 Nov 29; Vol. 11. Date of Electronic Publication: 2022 Nov 29.

Glial-Specific Deletion of Med12 Results in Rapid Hearing Loss via Degradation of the Stria Vascularis.

Autor: Huang TW; Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas 77030., Iyer AA; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas 77030.; Program in Genetics & Genomics, Baylor College of Medicine, Houston, Texas 77030., Manalo JM; Department of Biochemistry and Molecular Biology, The University of Texas Health Science Center at Houston, Houston, Texas 77030., Woo J; Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas 77030., Bosquez Huerta NA; Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas 77030.; Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030., McGovern MM; Department of Neuroscience, Baylor College of Medicine, Houston, Texas 77030., Schrewe H; Department of Developmental Genetics, Max Planck Institute for Molecular Genetics, 14195 Berlin, Germany., Pereira FA; Huffington Center on Aging, Baylor College of Medicine, Houston, Texas 77030.; Department of Otolaryngology, Baylor College of Medicine, Houston, Texas 77030., Groves AK; Department of Neuroscience, Baylor College of Medicine, Houston, Texas 77030.; Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas 77030.; Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030.; Program in Genetics & Genomics, Baylor College of Medicine, Houston, Texas 77030., Ohlemiller KK; Department of Otolaryngolgy, Central Institute for the Deaf, Fay and Carl Simons Center for Biology of Hearing and Deafness, Washington University in St. Louis, St. Louis, Missouri 63110., Deneen B; Center for Cell and Gene Therapy, Baylor College of Medicine, Houston, Texas 77030 deneen@bcm.edu.; Department of Neuroscience, Baylor College of Medicine, Houston, Texas 77030.; Program in Developmental Biology, Baylor College of Medicine, Houston, TX 77030.; Department of Neurosurgery, Baylor College of Medicine, Houston, Texas 77030.

Publikováno v: The Journal of neuroscience : the official journal of the Society for Neuroscience [J Neurosci] 2021 Aug 25; Vol. 41 (34), pp. 7171-7181. Date of Electronic Publication: 2021 Jul 12.

Avoiding exploitation in multinational covid-19 vaccine trials.

Autor: Iyer AA; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA.; Fogarty International Center, National Institutes of Health, Bethesda, MD, USA., Millum J; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA.; Fogarty International Center, National Institutes of Health, Bethesda, MD, USA., Grady C; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA.; Fogarty International Center, National Institutes of Health, Bethesda, MD, USA., Wendler D; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA.; Fogarty International Center, National Institutes of Health, Bethesda, MD, USA.

Publikováno v: BMJ (Clinical research ed.) [BMJ] 2021 Mar 04; Vol. 372, pp. n541. Date of Electronic Publication: 2021 Mar 04.

Patient and family social media use surrounding a novel treatment for a rare genetic disease: a qualitative interview study.

Autor: Iyer AA; Stanford Center for Biomedical Ethics, Department of Medicine, Stanford University, Stanford, CA, USA.; Department of Bioethics, National Institutes of Health Clinical Center, Bethesda, MD, USA., Barzilay JR; Stanford University School of Medicine, Stanford, CA, USA., Tabor HK; Stanford Center for Biomedical Ethics, Department of Medicine, Stanford University, Stanford, CA, USA. hktabor@stanford.edu.

Publikováno v: Genetics in medicine : official journal of the American College of Medical Genetics [Genet Med] 2020 Nov; Vol. 22 (11), pp. 1830-1837. Date of Electronic Publication: 2020 Jun 30.