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Alessandra Manfre's Fundraiser:

Sofia Sees Hope!

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Alessandra Manfre

THE STORY:

Laura Manfre wrote -

Sofia was told she would be totally blind someday. 

Sofia has Leber's Congenital Amaurosis, a rare genetic disorder.  Although she has some vision now, this is a degenerative condition that causes the cones and rods in her retina to deteriorate.  Eventually they will stop working altogether. 

Unless…

We can raise the money to fund the gene therapy for her gene.  (IQCB1, if you're into details.) 

In simple words that her very non-scientific parents can understand: 

  • Your donation will go towards funding the development of a vector for the IQCB1 gene.
  • The vector is more or less a virus (a good one, we like to think of it as a boat) that carries the good IQCB1 gene (a sailor) into the retina.
  • Once in there, the good gene swims around, replicates, and replaces the bad genes.
  • Vision is restored!

This type of gene therapy has been done before for patients with LCA, but with different gene types and it works!  It's really pretty cool. 

This research won’t help only Sofia, but will benefit all kids and adults with the IQCB1 defect.  Also, once the vector is developed there is the opportunity to use this for other genetic disorders. 

To read more about the Foundation for Retinal Research, click here: http://www.tfrr.org/

If you want to connect with us directly, send an email to sofiasees@gmail.com.

If you're donating, THANKS, and don't forget to do a corporate donation match if you can!

DONATE

To This Fundraiser

$345

MONEY RAISED
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  • Anonymous

    $150

  • Joan Misa

    $50

  • Laura Caine

    $20

  • Melle J

    $75

  • Mike Boosinger

    $50

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14% Raised of $2,500 Goal

Fundraise for this Campaign

The Team: $22,296 TOTAL RAISED SO FAR

JOIN THE TEAM
Fundraiser Title

Naomi Rodriguez

Amount Raised

$2,751

 

138% Raised of $2,000 Goal

Fundraiser Title

Cristina Manfre

Amount Raised

$2,700

 

180% Raised of $1,500 Goal

Fundraiser Title

Sally Klich

Amount Raised

$2,530

 

253% Raised of $1,000 Goal

Fundraiser Title

The Bertrams

Amount Raised

$1,655

 

166% Raised of $1,000 Goal

Fundraiser Title

Amy DiRico

Amount Raised

$1,040

 

104% Raised of $1,000 Goal

Fundraiser Title

Hollis Scoggin

Amount Raised

$975

 

98% Raised of $1,000 Goal

Fundraiser Title

Team Terrier

Amount Raised

$775

Fundraiser Title

Annette Tonti

Amount Raised

$650

Donor Comments

Mike Boosinger

Mike Boosinger

DONATION: $50

5 years ago

Melle J

Melle J

DONATION: $75

Wishing everyone best of luck with the fund raising. 5 years ago

Laura Caine

Laura Caine

DONATION: $20

Always donate for a good cause..good luck x 5 years ago

Joan Misa

Joan Misa

DONATION: $50

For everything you and your family has done for me. A small thank you. 5 years ago

Anonymous

ANONYMOUS

DONATION: $150

5 years ago