NCT05740761 (MECPer-3D) is a clinical trial of Gene editing in vitro in Rett Syndrome, sponsored by University of Siena.

Who is sponsoring NCT05740761?

NCT05740761 is sponsored by University of Siena.

What drugs are being tested in NCT05740761?

Interventions in NCT05740761: Gene editing in vitro.

What condition does NCT05740761 study?

NCT05740761 studies Rett Syndrome.

Is NCT05740761 still recruiting?

NCT05740761 is currently recruiting now. Last updated 20 August 2025.

Last reviewed 2025-08-20 · How we verify

NCT05740761: MECPer-3D

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Gene Editing as a Therapeutic Approach for Rett Syndrome

Recruiting now Last updated 20 August 2025

What this trial tests

trial testing Gene editing in vitro in Rett Syndrome in 40 participants. Currently enrolling.

Timeline

1 March 2021

Primary endpoint
1 November 2025

1 March 2026

Quick facts

Lead sponsor	University of Siena
Status	Recruiting now
Study type	OBSERVATIONAL
Enrollment	40
Start date	1 March 2021
Primary completion	1 November 2025
Estimated completion	1 March 2026
Sites	1 location across Italy

Drugs / interventions tested

Gene editing in vitro

Conditions studied

Rett Syndrome — all drugs for Rett Syndrome →

Sponsor

University of Siena

Who can join

6 Months and older, female only, with Rett Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

We designed the project to validate CRISPR/Cas9-based gene editing combined with AAV-based delivery for correction of the most common MECP2 mutations both in vitro and in vivo.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Rett Syndrome: The Emerging Landscape of Treatment Strategies.
Percy AK, Ananth A, Neul JL. · · 2024 · cited 20× · PMID 39251501 · DOI 10.1007/s40263-024-01106-y
In vivo LNP-CRISPR Approaches for the Treatment of Hemophilia.
Lee JH, Han JP. · · 2024 · cited 12× · PMID 38538969 · DOI 10.1007/s40291-024-00705-1
Nanoparticle-mediated delivery of non-viral gene editing technology to the brain.
Williams L, Larsen J. · · 2024 · cited 7× · PMID 38042249 · DOI 10.1016/j.pneurobio.2023.102547
Cell-Penetrating Peptides and CRISPR-Cas9: A Combined Strategy for Human Genetic Disease Therapy.
de Morais CCPL, Correia EM, Bonamino MH, Vasconcelos ZFM. · · 2024 · cited 6× · PMID 39276086 · DOI 10.1089/hum.2024.020
Emerging Perspectives on Prime Editor Delivery to the Brain.
BenDavid E, Ramezanian S, Lu Y, Rousseau J, et al · · 2024 · cited 4× · PMID 38931430 · DOI 10.3390/ph17060763
CRISPR-Cas technologies in neurodegenerative disorders: mechanistic insights, therapeutic potential, and translational challenges.
Yashooa RK, Nabi AQ, Smail SW, Azeez SS, et al · · 2025 · cited 3× · PMID 41674784 · DOI 10.3389/fneur.2025.1737468
Diet and Nutrients in Rare Neurological Disorders: Biological, Biochemical, and Pathophysiological Evidence.
Briglia M, Allia F, Avola R, Signorini C, et al · · 2024 · cited 3× · PMID 39339713 · DOI 10.3390/nu16183114
Integrating CRISPR/Cas technology with clinical trials: Principles, progress and challenges.
Yang P, Khoshandam M, Bhia I, Raji S, et al · · 2025 · PMID 41809894 · DOI 10.1016/j.ajps.2025.101068

Verify or expand the search:

Other recruiting trials for Rett Syndrome

Currently open trials in the same condition.

NCT06840496 — To Investigate the Efficacy of Treatment With Oral NA-921 (Bionetide) Versus Placebo in Females With Rett Syndrome · Phase 3 · recruiting
NCT07151950 — Obi Medical Robot: Evaluating Effectiveness Related to Usability · active not recruiting
NCT06856759 — Single-Dose AAV-MECP2 Safety/Tolerability and Efficacy in Rett Syndrome · EARLY_PHASE1 · recruiting
NCT07418905 — Technology-supported Motor Rehabilitation for People With Rett Syndrome · NA · active not recruiting
NCT06152237 — Safety and Efficacy of TSHA-102 in Pediatric Females With Rett Syndrome (REVEAL Pediatric Study) · Phase 1, PHASE2 · active not recruiting

Other University of Siena trials

Trials by the same sponsor.

NCT07053865 — Electromyographic Assessment of Sleep Bruxism in Patients With Stage III and IV Periodontitis · completed
NCT07053878 — Changes in Bruxism Activity After Periodontal Treatment in Patients With Periodontitis · NA · completed
NCT07018284 — Postoperative Pain Evaluation After Obturation With Different Sealers · NA · recruiting
NCT07019298 — Success of Endodontic Treatment With Different Obturation Techniques and Sealer Cements · NA · recruiting
NCT06923748 — Comparison of Two Interdental Devices for Peri-Implant Plaque Control · NA · completed

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT05740761 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by University of Siena
Last refreshed: 20 August 2025

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05740761.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing