Table 2 CSF neurotransmitter metabolites.

From: Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

Metabolite (normal range)

Baseline

Month 3

Month 6

Month 12

Month 24

BL1

BL2

HVA (218–852 nmol/L)

      

  Subject 1

<5

15

49

59

52

12

  Subject 2

18

10

66

56

52

73

  Subject 3

12

14

71

89

103

137

  Subject 4

10a

7

12

75

57

  Subject 5

12

11

76

70

59

  Subject 6

5

8

134

138

  Subject 7

37

38

172

146

5-HIAA (66–338 nmol/L)

  Subject 1

10

7

<5

<5

7

<5

  Subject 2

<5

<5

<5

<5

<5

8

  Subject 3

5

<5

<5

<5

7

24

  Subject 4

<5

<5

13

25

<5

  Subject 5

21

<5

<5

8

<5

  Subject 6

<5

5

12

6

  Subject 7

5

42

26

14

3-OMD (<100 nmol/L)

      

  Subject 1

605

600

504

549

875

443

  Subject 2

719

556

489

410

368

460

  Subject 3

378

358

443

413

303

432

  Subject 4

2104a

578

517

426

441

  Subject 5

343

529

379

632

625

  Subject 6

429

411

446

325

  Subject 7

928

769

558

556

  1. HVA homovanillic acid, 5-HIAA 5-hydroxyindoleacetic acid, 3-OMD 3-O-methyldopa.
  2. aSubject 4 was taking carbidopa-levodopa at the time of the first baseline lumbar puncture. The medication was tapered off prior to the day of surgery (2nd baseline lumbar puncture).