Gene therapy for X-linked retinoschisis in children and adolescents

Inclusion Criteria:

1. Fully understand the purpose and requirements of the trial, voluntarily participate in the clinical trial and sign the informed consent, and be able to cooperate with the testing required in various studies according to the requirements of the program;
2. Male, ≥5 years old and \< 18 years old (subject to the date of signing the informed consent);
3. Clinically diagnosed XLRS, identified by genetic testing as RS1 gene mutation, and do not carry other mutations causing ophthalmic genetic diseases;
4. Best corrected visual acuity (BCVA) of the study eye ≤63 letters (equivalent to Snellen 20/63); Best corrected visual acuity (BCVA) ≥34 letters (equivalent to Snellen 20/200).

Exclusion Criteria:

1. Carbonic anhydrase inhibitor treatment within 3 months;
2. The presence of other conditions in the study eye that may cause vision loss (e.g., atrophy of the optic nerve, advanced glaucoma, uveitis, the presence of vesicular splitting chambers or vesicular retinal detachment that may affect the subretinal injection operator);
3. Those who study the retinal observation and examination caused by lens, cornea or other refractive interstitial turbidity;
4. There are eye conditions affecting subretinal injection or eye conditions affecting the determination of the study endpoint;
5. Patients who had undergone internal eye surgery within 6 months;
6. Patients with AAV8 neutralizing antibody titer ≥1:1000;
7. Have previously received any gene therapy or cell therapy;
8. fertile subjects are unwilling to use contraception;
9. The presence of any of the following: an active infection that the investigator believes may affect the patient's participation in the study or affect the study results that requires systematic treatment; Hepatitis B surface antigen positive, hepatitis B virus deoxyribonucleic acid (HBV DNA) copy number \> ULN; Hepatitis C virus (HCV) antibody positive and HCV-RNA copy number \>ULN; Treponema pallidum antibody positive; Positive for human immunodeficiency virus (HIV) antibodies;
10. Malignant tumors (basal cell or squamous cell skin cancer) were diagnosed within 5 years prior to screening;
11. has or has had a systemic immune system disease;
12. Laboratory values considered to be clinically significant were abnormal: alanine and/or aspartate transaminase \>2.5×ULN, total bilirubin \>1.5×ULN, serum creatinine \>1.5×ULN, prothrombin time ≥1.5×ULN, activated partial thromboplastin time ≥1.5×ULN; (13) have a severe allergy or a known allergy to the drugs used in the treatment or examination in the study protocol, including the investigational drug allergy;

(14) Other situations that the investigator considers inappropriate for participation in this study.