Evidence map›Paper›PMID 41827217›Full record

ReviewJournal of clinical medicine2026

The New Wave of Gene and Cell Therapies Across Diseases.

Adrianna Rieske, Dagmara Grot, Cezary Tręda, Aneta Włodarczyk, Ewelina Stoczyńska-Fidelus, Maria Jaskólska, Piotr Rieske

Abstract readReview
In one paragraph

Review in Journal of clinical medicine, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Genome editing between wonder and rejection.Frontiers in bioengineering and biotechnology · 2026
    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

7 authors.

Adrianna RieskeDepartment of Research and Development, Personather Ltd., Inwestycyjna 7 St, 95-050 Konstantynow Lodzki, Poland.
Dagmara GrotDepartment of Tumor Biology, Medical University of Lodz, Zeligowskiego 7/9, 90-752 Lodz, Poland.ORCID 0000-0002-3588-9140
Cezary TrędaDepartment of Research and Development, Personather Ltd., Inwestycyjna 7 St, 95-050 Konstantynow Lodzki, Poland.
Aneta WłodarczykDepartment of Research and Development, Personather Ltd., Inwestycyjna 7 St, 95-050 Konstantynow Lodzki, Poland.
Ewelina Stoczyńska-FidelusDepartment of Molecular Biology, Medical University of Lodz, Zeligowskiego 7/9, 90-752 Lodz, Poland.
Maria JaskólskaDepartment of Tumor Biology, Medical University of Lodz, Zeligowskiego 7/9, 90-752 Lodz, Poland.
Piotr RieskeDepartment of Research and Development, Personather Ltd., Inwestycyjna 7 St, 95-050 Konstantynow Lodzki, Poland.ORCID 0000-0002-2970-0970

Funding

Medical University of Lodz 503/0-166-01/503-01-001
6 · The paper itself

Abstract

Recent years have seen rapid progress in biological treatments for genetic diseases, as well as conditions like type 1 diabetes that lack an obvious genetic component. The authors sought to explain why this progress has emerged at this particular moment. The best way to illustrate this is by showcasing a wide range of therapies targeting diverse diseases. This progress has been driven by technological advances in genetically modified CAR-T and CAR-NK cells (e.g., using CRISPR or transgenes), which have led to significant improvements in cancer therapy. A key trend now is the emergence of "off-the-shelf" approaches aimed at generating cellular therapies compatible with a range of recipients by mitigating alloreactivity and immune rejection. Different diseases impose distinct biological and logistical limitations; thus, treatment of each patient requires an appropriate strategy. Emerging advances include the modification of therapeutic cells, either ex vivo or in vivo. Current options for transgene delivery mainly comprise lipid nanoparticles (LNPs), adeno-associated virus (AAV) vectors, and lentiviral vectors. Researchers also focus on selecting suitable promoters for specific expression in selected cell types. Altogether, these advances have led to remarkable progress in treating various diseases in recent years. This publication discusses the development of biological therapies, with particular emphasis on cell and gene therapies, illustrated by viable examples across various disorders. It covers implemented solutions for several types of cancer, as well as selected hereditary diseases and syndromes, including Huntington's disease, carbamoyl phosphate synthetase 1 (CPS1) deficiency, hemiplegia, epidermolysis bullosa, chronic granulomatous disease, and congenital deafness. Emerging applications in heart diseases and diabetes are also summarized, along with therapeutic strategies involving tRNA gene editing. Although numerous strategies exist, only the most representative, practical, and up-to-date examples are emphasized.

Indexed as

CAR-Tcell therapiesCRISPRgene therapiesgenetic engineering

Identifiers

PMID41827217
PMCPMC12986383

What Socratic 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 Socratic graph.