ArticleNature medicine2026
AAV gene therapy for homozygous familial hypercholesterolemia: a phase 1 trial.
Article in Nature medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
1 citing paper in PubMed.
- Spatial Transcriptomics-Inspired Targeted Hydrogel for Dental Pulp Therapy via Strengthen Macrophage Communication.Advanced healthcare materials · 2026Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors.
Funding
Abstract
Homozygous familial hypercholesterolemia (HoFH) is a rare autosomal disease characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) levels and accelerated atherosclerotic cardiovascular disease. More than 80% of patients with HoFH carry low-density lipoprotein receptor (LDLR) mutations. Here we developed an adeno-associated virus gene therapy designed to express LDLR in the liver and evaluated its safety and efficacy in lowering LDL-C levels in experimental animals and in patients with HoFH. NGGT006, a recombinant adeno-associated virus serotype 8-based vector containing a codon-optimized LDLR complementary DNA, lowered LDL-C levels in Ldlr
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.