Leber Optic Hereditary Neuropathy
Conditions
Interventions
Group 1:Intravitreal injection of RAAV2-ND1 (1 × 109 Vg)
Group 2:Intravitreal injection of rAAV2-ND1 (1 × 1010 vg) 0.05ml
Sponsors
Taihe Hospital, Hubei University of Medicine, Shiyan, Hubei Province, China
Eligibility
Sex/Gender
All
Age
12 Years to 35 Years
Inclusion criteria
Inclusion criteria: 1. Met Leber's diagnostic criteria for hereditary optic neuropathy and was identified by gene sequencing as 3460 and other confirmed ND1 site mutations (3635 G A, 3733 G > A, 4136 A > G, 3571 C > T, 4171 C > A, 3697 G > A), without other treatment in the past 3 months and with no improvement in vision; 2. The fractional vision in both eyes was less than 0.3. 3. Aged >= 12 and <= 35 years, male or female, able to tolerate local anesthesia operation; 4. Consent not to use other drugs or treatments for the disease during the treatment period; 5. The patient and his legal representative participate voluntarily and sign the informed consent.
Exclusion criteria
Exclusion criteria: 1. Combined with the following diseases: 1) Chronic diseases such as diabetes; 2) Severe primary diseases such as cardiovascular, cerebrovascular, liver and kidney; 3) Other systemic neurological diseases; 4) Patients in convalescence after heart surgery, etc.; 5) Patients with mental disorders; 6) Patients with other eye diseases; 7) Patients with Leber Plus; 2. Patients who have participated in other clinical studies in recent 3 months; 3. Alcoholism, smoking, drug use or a history of drug abuse or exposure to toxic substances; 4. Pregnant women, lactating women or those who plan to become pregnant within 12 months, and male patients who plan to have a child within 2 years; 5. Other reasons why the researcher considers it inappropriate to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Best corrected visual acuity;visual field; | — |
Countries
China
Contacts
Public ContactBin Li
Taihe Hospital, Hubei University of Medicine, Shiyan, Hubei Province, China
Outcome results
None listed