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Table 2 Retinopathies represented in newspaper articles compared to phase of clinical trials registered in clinicaltrials.gov

From: Ocular gene transfer in the spotlight: implications of newspaper content for clinical communications

Diseaseb(number of articlesa)

Nature of retinopathy

Clinical trial number

Clinical trial phase

Gene targeted

Vector type

Number (n) and age of participants

Leber congential amaurosis (84)

Progressive retinopathy with severe visual problems beginning in infancy.

NCT00999609

III

RPE65

Adeno-associated viral vector; AAV2-hRPE65v2

n = 24; 3 years and older

NCT00749957

I/II

RPE65

Recombinant adeno-associated viral vector; rAAV2-CB-hRPE65

n = 12; 6 years and older

NCT01208389

I/II

RPE65

Adeno-associated viral vector; AAV2-hRPE65v2

n = 12; 8 years and older

NCT00643747

I/II

RPE65

Recombinant adeno-associated viral vector; rAAV 2/2.hRPE65p.hRPE65

n = 12; 5-30 years old

NCT01496040

I/II

RPE65

Recombinant adeno-associated viral vector; rAAV-2/4.hRPE65

n = 9; 6-50 years old

NCT00481546

I

RPE65

Recombinant adeno-associated viral vector; rAAV2-CBSB-hRPE65

n = 15; 8 years and older

NCT00821340

I

RPE65

Recombinant adeno-associated viral vector; rAAV2-hRPE65

n = 10; 8 years and older

NCT00516477

I

RPE65

Adeno-associated viral vector; AAV2-hRPE65v2

n = 12; 8 years and older

Retinitis pigmentosa (37)

Progressive retinopathy characterized by gradual peripheral vision loss

NCT01482195

I

MERTK

Recombinant adeno-associated viral vector; rAAV2-VMD2-hMERTK

n = 6; 14-70 years old

Age-related macular degeneration (24)

Progressive central vision loss usually occurring in older adults

NCT01301443

I

Genes that encode endostatin and angiostatin

Lentiviral vector; RetinoStat

n = 18; 50 years and older

NCT01678872

I

Genes that encode endostatin and angiostatin

Lentiviral vector; RetinoStat

n = 18; 50 years and older

Choroideremia (7)

Progressive retinopathy characterized by gradual peripheral vision loss

NCT01461213

I/II

REP1

Adeno-associated viral vector; AAV.REP1

n = 12; 18 years and older

Stargardt macular degeneration (7)

Progressive degeneration of the macula causing central vision loss beginning in childhood

NCT01367444

I/IIa

ABCA4

Lentiviral vector; StarGen

n = 28; 18 years and older

Usher syndrome (7)

Progressive retinopathy (retinitis pigmentosa) combined with hearing loss

NCT01505062

I/IIa

MYO7A

Lentiviral vector; UshStat

n = 18; 18 years and older

  1. aSome articles targeted more than a single retinopathy.
  2. bNote that 18 articles referred to gene transfer targeting color blindness, one referred to retinocshisis, and one referred to congenital stationary night blindness, even though no clinical trials for these conditions were registered in clinicaltrials.gov.