[HTML][HTML] Modelling mitochondrial disease in human pluripotent stem cells: what have we learned?

CL McKnight, YC Low, DA Elliott, DR Thorburn… - International Journal of …, 2021 - mdpi.com
Mitochondrial diseases disrupt cellular energy production and are among the most complex
group of inherited genetic disorders. Affecting approximately 1 in 5000 live births, they are …

[HTML][HTML] Inborn errors of metabolism: Lessons from iPSC models

R Escribá, R Ferrer-Lorente, Á Raya - Reviews in Endocrine and Metabolic …, 2021 - Springer
The possibility of reprogramming human somatic cells to pluripotency has opened
unprecedented opportunities for creating genuinely human experimental models of disease …

[HTML][HTML] Quantitative analysis of mitochondrial morphologies in human induced pluripotent stem cells for Leigh syndrome

F Meshrkey, AC Ayuso, RR Rao, S Iyer - Stem cell research, 2021 - Elsevier
Mitochondria are dynamic organelles with wide range of morphologies contributing to
regulating different signaling pathways and several cellular functions. Leigh syndrome (LS) …

[HTML][HTML] Generation and evaluation of isogenic iPSC as a source of cell replacement therapies in patients with Kearns Sayre syndrome

G Lester Sequiera, A Srivastava, KN Alagarsamy… - Cells, 2021 - mdpi.com
Kearns Sayre syndrome (KSS) is mitochondrial multisystem disorder with no proven
effective treatment. The underlying cause for multisystem involvement is the energy deficit …

[HTML][HTML] Exploiting hiPSCs in Leber's hereditary optic neuropathy (LHON): present achievements and future perspectives

C Peron, A Maresca, A Cavaliere, A Iannielli… - Frontiers in …, 2021 - frontiersin.org
More than 30 years after discovering Leber's hereditary optic neuropathy (LHON) as the first
maternally inherited disease associated with homoplasmic mtDNA mutations, we still …

[HTML][HTML] Advances in mt-tRNA mutation-caused mitochondrial disease modeling: patients' brain in a dish

S Povea-Cabello, M Villanueva-Paz… - Frontiers in …, 2021 - frontiersin.org
Mitochondrial diseases are a heterogeneous group of rare genetic disorders that can be
caused by mutations in nuclear (nDNA) or mitochondrial DNA (mtDNA). Mutations in mtDNA …

[HTML][HTML] iPSC culture expansion selects against putatively actionable mutations in the mitochondrial genome

M Kosanke, C Davenport, M Szepes, L Wiehlmann… - Stem Cell Reports, 2021 - cell.com
Therapeutic application of induced pluripotent stem cell (iPSC) derivatives requires
comprehensive assessment of the integrity of their nuclear and mitochondrial DNA (mtDNA) …

Development of an in vitro Model to Explore the Impact of mtDNA Mutations on the Metabolism and Epigenome of Myogenic Cells

BJ O'Callaghan - 2021 - discovery.ucl.ac.uk
The work presented in this thesis describes the successful establishment of an in vitro model
of mitochondrial disease using hiPSC technology and targeted differentiation towards …

[PDF][PDF] Deleciones en el DNA mitocondrial causantes del síndrome de Pearson: modelos celulares y aproximaciones terapéuticas.

S Emperador Ortíz, J Montoya Villarroya - core.ac.uk
Este trabajo ha sido financiado con fondo de los proyectos de investigación del Instituto de
Salud Carlos III FIS PI14/00005 y PI17/00021, del Gobierno de Aragón (Grupos …

Deleciones en el DNA mitocondrial causantes del síndrome de Pearson: modelos celulares y aproximaciones terapéuticas.

CJ Hernández Ainsa, S Emperador Ortíz… - zaguan.unizar.es
Las mitocondrias son el único orgánulo de las células animales que contiene su propio
DNA (DNA mitocondrial o mtDNA), una molécula circular de doble cadena que codifica 37 …