Evidence map›Paper›PMID 41972732›Full record

ReviewCells2026

CRISPR-Cas9 Therapeutics in Early Clinical Development: Delivery and Molecular Diagnostics.

Adrianna Rutkowska, Tadeusz Strózik, Tomasz Wasiak, Damian Ciunowicz, Natalia Kapelan, Natalia Szczepaniak, Juliusz Sosnowski, Weronika Goślińska, Jakub Bartkowiak, Agata Budny-Lewandowska and 6 more

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Cells, 2026. 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

16 authors.

Adrianna RutkowskaDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.ORCID 0000-0002-0973-6052
Tadeusz StrózikDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.ORCID 0000-0002-1397-7166
Tomasz WasiakDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.ORCID 0000-0002-4508-9534
Damian CiunowiczDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Natalia KapelanDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Natalia SzczepaniakDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Juliusz SosnowskiDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Weronika GoślińskaDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Jakub BartkowiakDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Agata Budny-LewandowskaDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Patrycja AntończykDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Maria MarkiewiczDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Piotr GustawDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.
Kamil FiliksDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.ORCID 0009-0002-4193-8997
Maria JaskólskaDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.ORCID 0009-0000-2607-1892
Ewelina Stoczyńska-FidelusDepartment of Molecular Biology, Chair of Medical Biology, Medical University of Lodz, Zeligowskiego 7/9 St., 90-752 Lodz, Poland.

Funding

LEK-AM Pharmaceutical Company Ltd 2024/ABM/05/KPO/KPOD.07.07-IW.07-0242/24-00Medical University of Lodz 503/0-154-01/503-01-001
6 · The paper itself

Abstract

CRISPR-Cas9 has progressed from an experimental tool to a therapeutic modality, marked by the first regulatory approvals of an ex vivo-edited autologous CD34+ hematopoietic stem cell product that induces fetal hemoglobin (CASGEVY/exa-cel). In this narrative review, we synthesize modality-specific molecular diagnostic strategies used across early CRISPR clinical translation. In parallel, early clinical experience has begun to demonstrate the feasibility of in vivo editing, including subretinal delivery for

Indexed as

CRISPR-Associated Protein 9Gene EditingGenetic TherapyAnimalsCRISPR-Cas SystemsHumansMiceCRISPR-Associated Protein 9clinical trialsCRISPR–Cas9delivery systemsgene therapygenome editingmolecular diagnostics

Identifiers

PMID41972732
PMCPMC13072328

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.