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Clinical Applications of Gene Therapy for Immuno-Deficiencies
Published in Yashwant Pathak, Gene Delivery, 2022
Khushboo Faldu, Sakshi Gurbani, Jigna Shah
Strategies employed to cure HIV are subdivided into two groups: “sterilizing cure” eradicates HIV-1 from the patients while “functional cure” controls HIV prognosis in terms of virus replication and CD4 T-lymphocytes in the absence of ART therapy. AlloHSCT in the absence of ART therapy has cured three patients globally that received stem cells from donors that carried the homozygous CCR5Δ32/Δ32 mutation [91, 92]. The patients suffered homological cancer that required alloHSCT [93]. Gene therapy with various strategies, like adoptive transfer of chimeric antigen receptor T cells or T cells with affinity-enhanced T cell receptors, fusion inhibitors, trans-dominant proteins, ribozymes, endoribonucleases, antisense strategies, combinatorial strategies, genome editing, epigenome editing, are being developed [93].
Genetics and genomics of idiopathic pulmonary fibrosis
Published in Muhunthan Thillai, David R Moller, Keith C Meyer, Clinical Handbook of Interstitial Lung Disease, 2017
Susan K Mathai, David A Schwartz
In terms of therapeutic implications of epigenetic findings in IPF, examples from oncology suggest that the epigenome could be a target for intervention. DNA methyltransferases have been approved for haematologic abnormalities such as myelodysplastic syndrome (89) and have been investigated as therapeutic options for solid malignancies (90). Generally, DNA methyltransferases lack genome location specificity, but locus-specific genome editing technologies are an active area of research (65,91,92), suggesting that epigenome editing could be a future therapeutic avenue in IPF.
Is Dupras and Bunnik’s Framework for Assessing Privacy Risks in Multi-Omic Research and Databases Still Too Exceptionalist?
Published in The American Journal of Bioethics, 2021
This being said, research on epigenomics including large epigenomic datasets and epigenome editing alike is but one kind of omics going currently rather unnoticed by normative analyses. Therefore, research that highlights the ethical importance of epigenomic data (Dupras and Bunnik 2021) and of epigenome editing (Alex and Winkler n.d; WHO Expert Advisory Committe on Developing Global Standards for Governance and Oversight of Human Genome Editing 2021; Zeps et al. 2021) is always at risk of reductionism and of replacing genomic exceptionalism, that is, “the view that genomic information deserves special attention in ethics guidelines, laws and politics” (Dupras and Bunnik 2021, 49) and the view of “reserving disproportionate ethical sensibility and scrutiny to these data types at the expense of others” (Dupras and Bunnik 2021, 58), with genomic–epigenomic exceptionalism, a view that places special attention to genomic and epigenomic data alike.
The Mutual Benefit of the Integration of Philosophy and Bioethics – Our Experience from an Interdisciplinary Research Project on (Epi-)Genome Editing
Published in The American Journal of Bioethics, 2022
Karla Karoline Sonne Kalinka Alex, Eva C. Winkler
While Blumenthal-Barby et al. (2022) point out that “[p]hilosopher bioethicists need to learn the culture of both worlds [philosophy and (medical) bioethics] and move between them — not a small feat,” we assume that this is still an underestimation of the challenges of moving between many more than two worlds. Thus, the philosophical research associate in the project we draw upon, as a recent philosophy graduate (pre-PhD) had to deeply dive into specific biological debates to understand what epigenome editing is, for being able to compare it to genome editing. By this, we have learned that ethically evaluating novel technologies about which there is not yet any ethical debate requires more time than contributing to existing bioethical debates about, e.g., germline genome editing, might. In addition, deeply diving into topics outside of philosophy, and of traditional bioethics is challenging not only because of different disciplinary “cultures,” but also because of the body of knowledge of the respective field of practice that one needs to learn, and master. Nevertheless, it is necessary to move between, and through these many disciplines to address bioethical questions arising not primarily from philosophical arguments in order to bring philosophical arguments, such as the nonidentity or intention considerations (see above), into bioethical debate. Understanding a problem from the perspective of many disciplines is very important as normative arguments can have far-reaching implications. However, in research ethics more so than in clinical ethics, this often requires an immersion into many more than two disciplines.
Ethical Framework for Next-Generation Genome and Epigenome Editing
Published in The American Journal of Bioethics, 2020
Kyoko Akatsuka, Mitsuru Sasaki-Honda, Tsutomu Sawai
We concur with his attempts to distinguish germline interventions along dimensions for evaluating ethical, legal, and social issues associated with each type of intervention. However, given the rapid progress in genome and epigenome editing technologies, the proposed framework may not prove robust for anticipated interventions. In the following sections, we argue for a modified framework for next-generation genome and epigenome editing by considering examples of anticipated germline interventions.