Evidence map›Paper›PMID 39717225›Full record

ArticleMolecular therapy. Methods & clinical development2024

Comparative assessment of the transduction efficiency and safety associated with the delivery of AAV9-GFP vector via lumbar puncture to cynomolgus macaques with and without anti-AAV9 pre-existing antibodies.

Ghiabe H Guibinga, Janet Do, Binh Chu, Yin Gu, Rie Kikkawa, Xiaoguang Li, Fatih Ozsolak, Timothy MacLachlan

Abstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.

0numbers the graph read from it
0cells of the map it votes in
2citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

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.

2 · The registry

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.

3 · Its place in the literature

Who cites it

2 citing papers in PubMed.

  1. Intra-CNS AAV9-Molecular therapy. Advances · 2026
    Article
  2. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Ghiabe H GuibingaNovartis Gene Therapies, San Diego, CA, USA.
Janet DoNovartis Gene Therapies, San Diego, CA, USA.
Binh ChuNovartis Gene Therapies, San Diego, CA, USA.
Yin GuNovartis Gene Therapies, San Diego, CA, USA.
Rie KikkawaPreclinical Safety (PCS), Novartis Biomedical Research, East Hanover, NJ, USA.
Xiaoguang LiBiologics Research Center (BRC), Novartis Biomedical Research, San Diego, CA, USA.
Fatih OzsolakNovartis Gene Therapies, San Diego, CA, USA.
Timothy MacLachlanPreclinical Safety (PCS), Novartis Biomedical Research, Cambridge, MA, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Administration of AAV-based gene therapies into the intra-cerebrospinal fluid (CSF) compartments via routes such as lumbar puncture (LP) has been implemented as an alternative to intravenous dosing to target the CNS regions. This route enables lower doses, decreases systemic toxicity, and circumvents intravascular pre-existing anti-AAV antibodies. In this study, AAV9-GFP vectors were administered via LP to juvenile cynomolgus macaques with and without pre-existing serum anti-AAV9 antibodies at a 5.0 × 10

Indexed as

AAV9biomarkersintra-CSF dosingliver enzymesneutralizing antibodynon-human primatespre-existing immunitytoxicitytransduction efficiency

Identifiers

PMID39717225
PMCPMC11664412

What OpenQuestion holds

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Registered trials

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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.