https://www.moonshotsforunicorns.org/community-events daily 0.75 2022-09-16 https://www.moonshotsforunicorns.org/community-events/dl3es1lg7vncgasjld7au2sp3krsuj monthly 0.5 2022-09-16 https://www.moonshotsforunicorns.org/community-events/the-unicorn-ball monthly 0.5 2022-09-16 https://www.moonshotsforunicorns.org/community-events/event-two-7y6j8 monthly 0.5 2022-09-16 https://www.moonshotsforunicorns.org/community-events/event-one-yecn7 monthly 0.5 2022-09-16 https://www.moonshotsforunicorns.org/home daily 1.0 2023-08-27 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/32b48c48-2402-4a3e-abb5-05fffd63a4c4/stars.png Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/041e80be-b84e-4417-a669-07aa77076ee9/IMG_0333.HEIC.jpg Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/32b48c48-2402-4a3e-abb5-05fffd63a4c4/stars.png Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/041e80be-b84e-4417-a669-07aa77076ee9/IMG_0333.HEIC.jpg Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/041e80be-b84e-4417-a669-07aa77076ee9/IMG_0333.HEIC.jpg Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/32b48c48-2402-4a3e-abb5-05fffd63a4c4/stars.png Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/ad7c217a-5f2a-4dc0-bad7-2a1b89f28dd6/IMG_0164.HEIC.jpg Cure Single Gene Disorders | Moonshots for Unicorns - PGAP3 What is PGAP3? PGAP3 is expressed in every cell in the body. When it's faulty, children can experience symptoms in many body systems. Almost all children with PGAP3 have low muscle tone and intellectual disability, and they lack expressive speech. Common Symptoms of PGAP3-CDG Congenital heart defects Increased risk of forming clots Developmental delays Cognitive impairment Low muscle tone Ataxia/poor balance Seizures Speech and vision problems Gastroesophageal reflux constipation Feeding difficulties https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/4325f8de-210e-4e44-91a9-66afd0b51c32/star+2.png Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/ad7c217a-5f2a-4dc0-bad7-2a1b89f28dd6/IMG_0164.HEIC.jpg Cure Single Gene Disorders | Moonshots for Unicorns - PGAP3 What is PGAP3? PGAP3 is expressed in every cell in the body. When it's faulty, children can experience symptoms in many body systems. Almost all children with PGAP3 have low muscle tone and intellectual disability, and they lack expressive speech. Common Symptoms of PGAP3-CDG Congenital heart defects Increased risk of forming clots Developmental delays Cognitive impairment Low muscle tone Ataxia/poor balance Seizures Speech and vision problems Gastroesophageal reflux constipation Feeding difficulties https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/ad7c217a-5f2a-4dc0-bad7-2a1b89f28dd6/IMG_0164.HEIC.jpg Cure Single Gene Disorders | Moonshots for Unicorns - PGAP3 What is PGAP3? PGAP3 is expressed in every cell in the body. When it's faulty, children can experience symptoms in many body systems. Almost all children with PGAP3 have low muscle tone and intellectual disability, and they lack expressive speech. Common Symptoms of PGAP3-CDG Congenital heart defects Increased risk of forming clots Developmental delays Cognitive impairment Low muscle tone Ataxia/poor balance Seizures Speech and vision problems Gastroesophageal reflux constipation Feeding difficulties https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/4325f8de-210e-4e44-91a9-66afd0b51c32/star+2.png Cure Single Gene Disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://www.moonshotsforunicorns.org/meet-lucy daily 0.75 2024-09-23 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/10241e70-e694-4971-a81a-832edb69743b/IMG_2293.jpg Meet Lucy - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/10241e70-e694-4971-a81a-832edb69743b/IMG_2293.jpg Meet Lucy - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/10241e70-e694-4971-a81a-832edb69743b/IMG_2293.jpg Meet Lucy - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/758515a8-a706-4572-9553-18931cfe2ab8/IMG_0403.HEIC.jpg Meet Lucy - “We are still defiant, and there is good reason to believe there is a lot of hope for Lucy.” Unfortunately, the genetic diagnosis is true. Equally unfortunately, given that so few people have this genetic disorder, there are no trials anywhere in the world for PGAP3 and there is no active research on developing a therapy for children with PGAP3. However, we are still defiant, and there is good reason to believe there is a lot of hope for Lucy. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/758515a8-a706-4572-9553-18931cfe2ab8/IMG_0403.HEIC.jpg Meet Lucy - “We are still defiant, and there is good reason to believe there is a lot of hope for Lucy.” Unfortunately, the genetic diagnosis is true. Equally unfortunately, given that so few people have this genetic disorder, there are no trials anywhere in the world for PGAP3 and there is no active research on developing a therapy for children with PGAP3. However, we are still defiant, and there is good reason to believe there is a lot of hope for Lucy. One of her copies of PGAP3 is a new variant, which may be slightly milder than cases described previously. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/758515a8-a706-4572-9553-18931cfe2ab8/IMG_0403.HEIC.jpg Meet Lucy - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/3d4bfa26-ba71-4d1d-b290-6cf5ea497f35/IMG_0905.JPG.jpg Meet Lucy - “The sky is the limit to what we can do for her.” Few children with this disorder have ever been diagnosed so young or had such a mild initial presentation. Most importantly, Lucy is young, and as my grandfather would say, there is light behind those eyes – she is curious, loving, chatty, and has not yet developed any of the more serious features of this syndrome. She is truly an N-of-1, with a novel variant and a novel set of life circumstances. No child in the world with her condition has had the opportunity to have an effective treatment so early in life, which means that the sky is the limit to what we can do for her. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/3d4bfa26-ba71-4d1d-b290-6cf5ea497f35/IMG_0905.JPG.jpg Meet Lucy - “The sky is the limit to what we can do for her.” Few children with this disorder have ever been diagnosed so young or had such a mild initial presentation. Most importantly, Lucy is young, and as my grandfather would say, there is light behind those eyes – she is curious, loving, chatty, and has not yet developed any of the more serious features of this syndrome. She is truly an N-of-1, with a novel variant and a novel set of life circumstances. No child in the world with her condition has had the opportunity to have an effective treatment so early in life, which means that the sky is the limit to what we can do for her. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/3d4bfa26-ba71-4d1d-b290-6cf5ea497f35/IMG_0905.JPG.jpg Meet Lucy - “The sky is the limit to what we can do for her.” Few children with this disorder have ever been diagnosed so young or had such a mild initial presentation. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/cfe45ad4-d339-439c-90d6-c101407f47da/IMG_4308.HEIC.jpg Meet Lucy - “No pharma company, government, or investor is interested in funding this research – it has to be us, together with you.” Because there are simply not enough kids known to have Lucy’s genetic disorder, no pharma company, government, or investor is interested in funding this research – it has to be us, together with you. We are tackling her treatment from multiple angles - supplements, drug repurposing, and gene therapy. The work done for her treatment will hopefully lead to treatments for the dozens of children suffering from this condition around the world. Please help us fund this research and give Lucy and other children with PGAP3 the gifts of speech, walking, and a life without seizures. - Geri Landman | Co-founder, Pediatrician, and Mother https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/cfe45ad4-d339-439c-90d6-c101407f47da/IMG_4308.HEIC.jpg Meet Lucy - “No pharma company, government, or investor is interested in funding this research – it has to be us, together with you.” Because there are simply not enough kids known to have Lucy’s genetic disorder, no pharma company, government, or investor is interested in funding this research – it has to be us, together with you. We are tackling her treatment from multiple angles - supplements, drug repurposing, and gene therapy. Lucy will be the N-of-1, but the work done for her treatment will hopefully lead to treatments for the dozens of children suffering from this condition around the world. Please help us fund this research and give Lucy the gifts of speech, walking, and a life without seizures. - Geri Landman | Co-founder, Pediatrician, and Mother https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/cfe45ad4-d339-439c-90d6-c101407f47da/IMG_4308.HEIC.jpg Meet Lucy - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/88c88922-cd5f-47fb-b9e5-e42b08149f86/IMG_8270.jpg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/703c4015-a80a-45d5-ba6e-e397a82b4756/kreephotography047+%281%29.JPEG https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a3b8b664-8062-4226-8e3e-2b23f957dad0/IMG_8369.jpg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/7daa46f3-f108-4798-8534-11ae311b3b5e/IMG_8434.jpg https://www.moonshotsforunicorns.org/team daily 0.75 2025-09-22 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/dd54e49e-5307-4b93-a32f-c64e8ed602b2/John+Doyle.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/d2507b68-9e67-4edc-9163-7975b799c632/unnamed+%284%29.png Leadership, Scientists, and Clinicians | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1655770820977-1EYSN380M84FEG7DZE6B/image%2B%25287%2529.jpg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/bdbed5f6-fd1e-4e94-aac2-afa3e42ba1a1/image0+%289%29.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/2822cfc3-6913-4da9-a168-da4d41426301/Geri+headshot.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658201999621-UV096MJFQ94KQ90IKUZM/Geri+headshot.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658202701145-WRMMO605PUYGT3OBPLF5/amanda.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658204157866-5SVTU9JUMPYP26JRYGGX/79664781_10214000195793805_5911073608704196608_n.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658206778231-LJ54DO976O250A8PTN4R/cat-lutz-in-lab-at-bench.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658207861481-T2QCBL88TBT65GELN1YK/murray_stephen_w5a7787.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658208323561-54K20ZAL5DI7RTH9CBAZ/Eva-Morava-Kozicz-20751516.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1658215213017-TS7U2QRFQX880BITU5GQ/leanne.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/48d5ef54-3717-49ea-97dc-f6568d66664e/Ariel+Peters+Photo+%281%29.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/287fee41-8421-4762-bb46-e6b8b30efb0b/Headshot+-+J+Wetzel+%281%29.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/33836652-a9fc-4ac3-b35c-8ea06dc366be/soussan+n+%281%29.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/be9f360c-8b21-4d4d-b623-452c209252e1/ZLandmanMD.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/b35ea0de-d7c2-46ba-8ed0-d2aa3caa0ca1/matt+simon.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/c17d6e22-35e0-4152-baac-14084e805e69/ahrens-nicklas-rebecca.jpg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/d5262173-5897-4ad1-a44f-f05810f41247/fyodor_urnov.jpg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/51637f72-6bb1-4e34-9eda-f05f9e260a40/thyago.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/381e1abd-7a56-498b-871e-1c5185b4b3d9/ilana.jpeg Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/e373a33a-bd57-4a8d-a0fc-1b0384a2e828/Alex+Fay.webp Leadership, Scientists, and Clinicians | Moonshots for Unicorns https://www.moonshotsforunicorns.org/updates daily 0.75 2025-09-22 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1768b0cc-3c90-4aa4-a004-a108ec4f51d6/IMG_0434.HEIC.jpg Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a83806df-4b80-43e4-9b16-97eae9273613/IMG_1310.jpg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/f1fad3f4-8424-424f-aee9-6969a1c888a0/Screenshot+2024-09-23+at+3.30.36%E2%80%AFPM.png https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/e345c092-54e3-4c8a-84d9-88bf9cfa5b03/IMG_7180.jpg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/238163f7-7100-4402-b53a-c798bd8f5881/Screenshot+2024-09-23+at+4.19.21%E2%80%AFPM.png https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/87604453-cf8c-469e-b162-70e9136ccd44/Screen+Shot+2023-08-24+at+10.11.08+AM.png https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/03bc10ad-7983-4991-8795-12abc0d978c4/Screen+Shot+2023-04-23+at+9.25.47+PM.png https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/e197e531-b2ad-4541-816e-2558a9256f77/Screen+Shot+2022-11-06+at+7.56.11+AM.png https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/c656292e-f7aa-46ce-a978-5d5960a2ffbe/Per1_deletion_growthrate_validation_220804.png https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/5adc951c-b5fc-48e4-8fb9-ff006cb7d985/Jax.jpeg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/93f6cba8-2a62-41d2-a641-f1545ee612af/stars.png Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a330c44f-71f8-4f4d-b854-8fbef021a509/Screen+Shot+2022-07-02+at+1.22.20+PM.png Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/9b854f8c-a485-4c50-b47b-03ab224b828c/Screen+Shot+2022-07-02+at+1.22.20+PM.png Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/93f6cba8-2a62-41d2-a641-f1545ee612af/stars.png Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/9b854f8c-a485-4c50-b47b-03ab224b828c/Screen+Shot+2022-07-02+at+1.22.20+PM.png Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/93f6cba8-2a62-41d2-a641-f1545ee612af/stars.png Scientific Updates - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://www.moonshotsforunicorns.org/donate daily 0.75 2024-09-27 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a87b4ac0-f392-43cd-a99e-91b126d46831/IMG_0392.HEIC.jpg Donate to cure single-gene disorders | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/93f6cba8-2a62-41d2-a641-f1545ee612af/stars.png Donate to cure single-gene disorders | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://www.moonshotsforunicorns.org/aboutmoonshots daily 0.75 2023-08-27 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/79fd870c-ead9-4499-810e-959f59f5951a/IMG_5189.jpg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/e4c6dc1b-fc9b-47bc-b3ae-63d853694cc8/Screen+Shot+2023-08-26+at+10.40.54+PM.png https://www.moonshotsforunicorns.org/unicorn-crusader daily 0.75 2022-07-23 https://www.moonshotsforunicorns.org/news daily 0.75 2024-03-01 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a330c44f-71f8-4f4d-b854-8fbef021a509/Screen+Shot+2022-07-02+at+1.22.20+PM.png In the news | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/93f6cba8-2a62-41d2-a641-f1545ee612af/stars.png In the news | Moonshots for Unicorns - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1660954956771-ZFVMXQKXIYNXPGJD750X/IMG_4028.jpg In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1660955211314-Q6C5VQZ5BPFUNV61CCCN/Screen+Shot+2022-08-19+at+5.26.10+PM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1660955479219-GAMDWOVDGBPV77BGVHIO/Screen+Shot+2022-08-19+at+5.26.29+PM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1660955629537-UM56G55OQXO8L4F61YPJ/Screen+Shot+2022-08-19+at+5.33.30+PM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1661561140506-8ZNA442FFJT0MBFZRCDY/Screen+Shot+2022-08-26+at+5.45.25+PM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1662699183309-TZQF8AM84UCFW4LKTOEG/Screen+Shot+2022-09-08+at+9.52.43+PM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1662699726754-2WT5PNM8IETFZBEWHIMP/Screen+Shot+2022-09-08+at+10.01.44+PM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/8cdd76af-2b71-4928-93e4-30d30f994b57/Screen+Shot+2022-09-25+at+8.59.45+AM.png In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/c42fc078-3b6b-4b1a-a575-52943441f201/ratio3x2_1200.jpeg In the news | Moonshots for Unicorns https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/1e7a12aa-787d-4524-9f54-10ba8b9d6d91/Screenshot+2024-02-29+at+8.49.45+PM.png In the news | Moonshots for Unicorns https://www.moonshotsforunicorns.org/pies-for-pgap daily 0.75 2022-08-18 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/18a80377-f2c5-4e7a-b022-cb1d6ecd8734/pie.jpg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a330c44f-71f8-4f4d-b854-8fbef021a509/Screen+Shot+2022-07-02+at+1.22.20+PM.png Pies for PGAP - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/93f6cba8-2a62-41d2-a641-f1545ee612af/stars.png Pies for PGAP - Make it stand out Whatever it is, the way you tell your story online can make all the difference. https://www.moonshotsforunicorns.org/pgap3yeast01 daily 0.75 2022-08-27 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/a013c727-21af-4b3a-80c5-af24e0316fac/Per1_deletion_growthrate_validation_220804.png pgap3yeast01 Per1∆ yeast strains have been thawed. The homozygous diploid strain has not been able to grow yet. However both mating types of the haploid deletion strains, as well as the heterozygous diploid strain (one functional copy of Per1), are growing successfully. Encouragingly, both haploid mating types of Per1∆ yeast exhibit significantly attenuated growth compared to the heterozygote with functional Per1 (see attached image). This is an excellent sign that Per1∆ exhibits a rescuable phenotype. Next week the yeast Cure Guides will troubleshoot the growth of the diploid knockout strain and test the sensitivity of the Per1 deletion strains to heat stress, which the literature indicates will further widen the gap between Per1∆ and Per1 +/- growth rates. In parallel, we’re in discussion with UCSF’s small molecule discovery center to ensure that your screens are charged at the external non-profit rate (as opposed to the external for-profit rate, which is higher). I’ll also be meeting with the yeast Cure Guides on Wednesday morning next week, so if you have any specific questions for them, please do share them with me and I will pass them along. Have a great weekend! Justin https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/b3e1d1a7-7f03-4174-8892-9faf45636d09/20220810_per1del_OD_growthrate_37C.png pgap3yeast01 To date, we have successfully validated a growth defect in Per1∆ yeast at 30ºC and 37ºC. This growth rate was measured using two orthogonal methods (OD and the BacTiter-Glo luminescence assay). The X-axis represents the initial cell count, and the Y-axis represents the initial output. See below for a legend identifying the different strains: BBY4741: Wild-type yeast (baseline) YSC6272: MATalpha Per1∆ (deficient) YSC6273: MATa Per1∆ (deficient) YSC6274: Heterozygous diploid Per1∆ (control, one functional Per1 copy) YSC6275: Homozygous diploid Per1∆ (deficient) It is encouraging to see the growth defect even under unstressed conditions (30ºC). With the validated strains and assay in-hand, we are on track to begin screening in late August. Additionally, last week I told you that we were working with UCSF's core facility to be billed at the external non-profit rate for the screen. I'm encouraged to report that we received confirmation that we will be charged at this rate. We will begin setting up a contract on Monday. Lastly, on the Lucy avatar front, we completed sequence drafting earlier today. These sequences will be used to generate mutant yeast. Once we confirm them internally, we will place an order to have them synthesized. Twist estimates that we will receive these sequences before Labor Day, but in my experience it's best to expect that they may take a little longer, as things can go wrong in the synthesis process (up to ~1 additional week or so). Our goal is to be ready to screen these lines by mid-September, but this will be somewhat dependent on Twist. I will keep you posted as things progress here. Let me know if you'd like to discuss the data any further! Have a great weekend, Justin https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/fdf8fc27-48ac-4099-a2b6-3a95663d8352/20220810_per1del_OD_growthrate_30C.png pgap3yeast01 To date, we have successfully validated a growth defect in Per1∆ yeast at 30ºC and 37ºC. This growth rate was measured using two orthogonal methods (OD and the BacTiter-Glo luminescence assay). The X-axis represents the initial cell count, and the Y-axis represents the initial output. See below for a legend identifying the different strains: BBY4741: Wild-type yeast (baseline) YSC6272: MATalpha Per1∆ (deficient) YSC6273: MATa Per1∆ (deficient) YSC6274: Heterozygous diploid Per1∆ (control, one functional Per1 copy) YSC6275: Homozygous diploid Per1∆ (deficient) It is encouraging to see the growth defect even under unstressed conditions (30ºC). With the validated strains and assay in-hand, we are on track to begin screening in late August. Additionally, last week I told you that we were working with UCSF's core facility to be billed at the external non-profit rate for the screen. I'm encouraged to report that we received confirmation that we will be charged at this rate. We will begin setting up a contract on Monday. Lastly, on the Lucy avatar front, we completed sequence drafting earlier today. These sequences will be used to generate mutant yeast. Once we confirm them internally, we will place an order to have them synthesized. Twist estimates that we will receive these sequences before Labor Day, but in my experience it's best to expect that they may take a little longer, as things can go wrong in the synthesis process (up to ~1 additional week or so). Our goal is to be ready to screen these lines by mid-September, but this will be somewhat dependent on Twist. I will keep you posted as things progress here. Let me know if you'd like to discuss the data any further! Have a great weekend, Justin https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/e59a3b84-6d12-4856-a787-574b9c08fa5f/20220810_per1del_bactiterglo_growthrate_37C.png pgap3yeast01 To date, we have successfully validated a growth defect in Per1∆ yeast at 30ºC and 37ºC. This growth rate was measured using two orthogonal methods (OD and the BacTiter-Glo luminescence assay). The X-axis represents the initial cell count, and the Y-axis represents the initial output. See below for a legend identifying the different strains: BBY4741: Wild-type yeast (baseline) YSC6272: MATalpha Per1∆ (deficient) YSC6273: MATa Per1∆ (deficient) YSC6274: Heterozygous diploid Per1∆ (control, one functional Per1 copy) YSC6275: Homozygous diploid Per1∆ (deficient) It is encouraging to see the growth defect even under unstressed conditions (30ºC). With the validated strains and assay in-hand, we are on track to begin screening in late August. Additionally, last week I told you that we were working with UCSF's core facility to be billed at the external non-profit rate for the screen. I'm encouraged to report that we received confirmation that we will be charged at this rate. We will begin setting up a contract on Monday. Lastly, on the Lucy avatar front, we completed sequence drafting earlier today. These sequences will be used to generate mutant yeast. Once we confirm them internally, we will place an order to have them synthesized. Twist estimates that we will receive these sequences before Labor Day, but in my experience it's best to expect that they may take a little longer, as things can go wrong in the synthesis process (up to ~1 additional week or so). Our goal is to be ready to screen these lines by mid-September, but this will be somewhat dependent on Twist. I will keep you posted as things progress here. Let me know if you'd like to discuss the data any further! Have a great weekend, Justin https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/81bc4e5a-8be3-4a69-9e27-0c1303560112/20220810_per1del_bactiterglo_growthrate_30C.png pgap3yeast01 To date, we have successfully validated a growth defect in Per1∆ yeast at 30ºC and 37ºC. This growth rate was measured using two orthogonal methods (OD and the BacTiter-Glo luminescence assay). The X-axis represents the initial cell count, and the Y-axis represents the initial output. See below for a legend identifying the different strains: BBY4741: Wild-type yeast (baseline) YSC6272: MATalpha Per1∆ (deficient) YSC6273: MATa Per1∆ (deficient) YSC6274: Heterozygous diploid Per1∆ (control, one functional Per1 copy) YSC6275: Homozygous diploid Per1∆ (deficient) It is encouraging to see the growth defect even under unstressed conditions (30ºC). With the validated strains and assay in-hand, we are on track to begin screening in late August. Additionally, last week I told you that we were working with UCSF's core facility to be billed at the external non-profit rate for the screen. I'm encouraged to report that we received confirmation that we will be charged at this rate. We will begin setting up a contract on Monday. Lastly, on the Lucy avatar front, we completed sequence drafting earlier today. These sequences will be used to generate mutant yeast. Once we confirm them internally, we will place an order to have them synthesized. Twist estimates that we will receive these sequences before Labor Day, but in my experience it's best to expect that they may take a little longer, as things can go wrong in the synthesis process (up to ~1 additional week or so). Our goal is to be ready to screen these lines by mid-September, but this will be somewhat dependent on Twist. I will keep you posted as things progress here. Let me know if you'd like to discuss the data any further! Have a great weekend, Justin https://www.moonshotsforunicorns.org/upcoming-events daily 0.75 2022-09-11 https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/9046ef3a-cac7-4401-aef2-25ff3b80d9dc/My+Invitation.jpeg https://images.squarespace-cdn.com/content/v1/628fd4fe1178c71e6efe2de9/68830c79-9057-40fd-9d1c-27a0d9a313c3/person-sean-george.jpeg