A retrograde approach for liver gene transfer

Brunetti‐Pierri, Nicola; Gissen, Paul

doi:10.1016/j.omtm.2022.11.002

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Three Decades Of Gene Therapy For Otcd1

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2023

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“…By aiming for a further dose reduction, the same vector construct was delivered through a hydrodynamic injection technique and maintained phenotypic correction at a lower dose of 5 × 10 11 particles/kg [ 41 ]. However, this type of hydrodynamic injection is unsuitable for humans, and no further translational work with Ads was undertaken in OTCD [ 42 , 43 , 44 ].…”

Section: Three Decades Of Gene Therapy For Otcdmentioning

confidence: 99%

Genetic Therapy Approaches for Ornithine Transcarbamylase Deficiency

Yılmaz

Gissen

2023

Biomedicines

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Ornithine transcarbamylase deficiency (OTCD) is the most common urea cycle disorder with high unmet needs, as current dietary and medical treatments may not be sufficient to prevent hyperammonemic episodes, which can cause death or neurological sequelae. To date, liver transplantation is the only curative choice but is not widely available due to donor shortage, the need for life-long immunosuppression and technical challenges. A field of research that has shown a great deal of promise recently is gene therapy, and OTCD has been an essential candidate for different gene therapy modalities, including AAV gene addition, mRNA therapy and genome editing. This review will first summarise the main steps towards clinical translation, highlighting the benefits and challenges of each gene therapy approach, then focus on current clinical trials and finally outline future directions for the development of gene therapy for OTCD.

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