Key result
Autologous implantation of LCAT-GMACs in a patient with familial LCAT deficiency was safe and increased serum LCAT activity by approximately 50% of baseline, sustaining this increase over three years.
Why the study?
Familial LCAT deficiency is a severe inherited disease without effective treatment, characterized by severe low HDL, corneal opacity, hemolytic anemia, and renal injury.
Does autologous implantation of LCAT-GMACs improve clinical and biochemical outcomes in a patient with familial LCAT deficiency?
Case Report (n=1)
Open-label
No
Does autologous implantation of LCAT-GMACs improve clinical and biochemical outcomes in a patient with familial LCAT deficiency?
First-in-human autologous implantation of LCAT-expressing genetically modified adipocytes was safe and partially effective for treating anemia and proteinuria in a patient with familial LCAT deficiency.
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Supports exploration of LCAT-GMAC therapy in LCAT deficiency; leaves open clinical efficacy pending controlled trials.
Aso et al. (2022) conducted a case report in Familial LCAT deficiency (n=1). LCAT-GMAC (genetically modified adipocytes secreting LCAT) vs. Baseline was evaluated on Safety and adverse-event profiles. Autologous implantation of LCAT-GMACs in a patient with familial LCAT deficiency was safe and increased serum LCAT activity by approximately 50% of baseline, sustaining this increase over three years.
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