Evidence map›Paper›PMID 39651221›Full record

ArticlebioRxiv : the preprint server for biology2024

Engineered CRISPR-Base Editors as a Permanent Treatment for Familial Dysautonomia.

Shuqi Yun, Anil Chekuri, Jennifer Art, Krishnakanth Kondabolu, Susan A Slaugenhaupt, Nadja Zeltner, Benjamin P Kleinstiver, Elisabetta Morini, Christiano R R Alves

Abstract readPreprint
In one paragraph

Article in bioRxiv : the preprint server for biology, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing 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

0 citing papers in PubMed.

No citing paper in PubMed yet.

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

9 authors.

Shuqi YunCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.ORCID 0009-0006-2228-6515
Anil ChekuriCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.ORCID 0000-0002-0652-2630
Jennifer ArtCenter for Molecular Medicine, University of Georgia, Athens, GA, USA.ORCID 0000-0002-2470-688X
Krishnakanth KondaboluCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.
Susan A SlaugenhauptCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.
Nadja ZeltnerCenter for Molecular Medicine, University of Georgia, Athens, GA, USA.
Benjamin P KleinstiverCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.ORCID 0000-0002-5469-0655
Elisabetta MoriniCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.ORCID 0000-0002-1658-5213
Christiano R R AlvesCenter for Genomic Medicine, Massachusetts General Hospital Research Institute, Boston, MA, USA.ORCID 0000-0002-2646-9689

Funding

PROJECT 4: Somatic evolution of the hematopoietic system in cardiovascular diseaseP01HL142494 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Benjamin Peter Kleinstiver · 2019 to 2026
$19.5M
Modeling and Therapeutic Approaches for Genetic VasculopathiesR01NS125353 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI MARK E LINDSAY, Patricia L Musolino · 2022 to 2026
$3.5M
Scalable Development of Custom Genome Editing TechnologiesDP2CA281401 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI KLEINSTIVER, BENJAMIN PETER · 2022 to 2025
$2.5M
Understanding Neuronal Cell type-Specific Dysregulation in Familial DysautonomiaR01NS095640 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI Elisabetta Morini, Susan A Slaugenhaupt · 2024 to 2026
$1.9M
Developing Splicing-Targeted Therapeutic Strategies for Neurological DiseasesR01NS124561 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI MORINI, ELISABETTA, TELO BAPTISTA LIMA DA SILVA, MARIA CATARINA · 2022 to 2025
$1.7M
Development of in Vivo Base Editing as a Genetic Treatment for Spinal Muscular AtrophyK01NS134784 · NINDS · UNIVERSITY OF PITTSBURGH AT PITTSBURGH · PI Christiano Alves · 2024 to 2026
$726k
A one and done therapeutic strategy to correct ELP1 splicing defect in familial dysautonomiaR21EY037018 · NEI · MASSACHUSETTS GENERAL HOSPITAL · PI Christiano Alves, Elisabetta Morini · 2025 to 2026
$454k
NCI NIH HHS DP2 CA281401NEI NIH HHS R21 EY037018NHLBI NIH HHS P01 HL142494NINDS NIH HHS K01 NS134784NINDS NIH HHS R01 NS095640NINDS NIH HHS R01 NS124561NINDS NIH HHS R01 NS125353
6 · The paper itself

Abstract

Familial dysautonomia (FD) is a fatal autosomal recessive congenital neuropathy caused by a T-to-C mutation in intron 20 of the

Indexed as

bystander editingCas9CRISPRELP1genome editingneurodegenerative diseasesplicing

Identifiers

PMID39651221
PMCPMC11623606

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

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.