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Sickle Cell Clinical Trial Aims to Cure Disease by Correcting Patient’s Mutated Gene

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

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Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.
Brooklynn Haynes is participating in a research trial that aims to cure sickle cell anemia, a disease that affects 100,000 people in the U.S. Photo courtesy of UCSF.

Benioff Children’s Oakland Receives $17M in Grants to Launch 4-Year Study Using CRISPR to Eradicate Sickle Cell Mutation

By Lorna Fernandes

UCSF Benioff Children’s Hospital Oakland has received the largest research trial grant in its history to launch an innovative clinical trial that aims to cure sickle cell disease.

The study, which plans to enroll its first patient by the middle of 2022, will use CRISPR-Cas9 gene-editing technology on patients’ own blood stem cells to correct the mutated gene that causes the debilitating disorder.

Benioff Oakland researchers have received $17 million to support the 4-year trial, in two grants of $8.4 million from the California Institute for Regenerative Medicine (CIRM) and $8.6 million from the NIH Cure Sickle Cell Initiative, supported by the National Heart, Lung, and Blood Institute (NHLBI).

This trial is the first to apply CRISPR-Cas9 technology in humans using a non-viral therapy to correct the mutation that causes sickle cell disease and is part of a UC research consortium led by UCSF with UCLA and UC Berkeley.

Researchers are recruiting patients for treatment in Oakland and Los Angeles, beginning with up to six adults with sickle cell disease. If found to be safe and effective, it will expand to enroll three adolescents aged 12 to 17 years old. Seven patients are expected to be treated in Oakland and two at UCLA.

It is the first-time clinical researchers have attempted to correct a harmful beta-globin gene mutation in a patient’s own cells with non-virally delivered CRISPR gene-correction tools, according to Mark Walters, MD, a professor of pediatrics at UCSF and principal investigator of the clinical trial and gene editing project.

“This therapy has the potential to transform sickle cell disease care by producing an accessible, curative treatment that is safer than the current therapy of stem cell transplant from a healthy bone marrow donor,” Walters said. “If this is successfully applied in young patients, it has the potential to prevent irreversible complications of the disease.”

Sickle cell disease is a genetic blood disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently, and progresses to a chronic vascular disease that affects approximately 100,000 Americans and millions worldwide, with a disproportionate effect on the Black community.

This clinical study continues BCH Oakland’s commitment to treating and finding a cure for sickle cell disease. The Comprehensive Sickle Cell Disease Center was created at the hospital in 1973 to provide expert diagnosis, treatment and long-term health management for children and adults with sickle cell disease.

Now known as the BCH Oakland Sickle Cell Center of Excellence, it is recognized as having one of the largest and most comprehensive sickle cell programs in the Western United States.

This trial will combine CRISPR technology developed at Innovative Genomics Institute (IGI) – a joint UC Berkeley-UCSF initiative founded by Nobel Laureate Jennifer Doudna – with UCLA’s expertise in genetic analysis and cell manufacturing, as well as its clinical excellence in the field, and nearly 50 years of expertise at Benioff Children’s Oakland in sickle cell care, including cord blood and marrow transplantation, and gene therapy.

“The use of CRISPR gene editing to fix the mutation causing sickle cell disease in each patient’s own blood-forming stem cells required the development of new methods to produce more than 100 million cells per patient that are gene-corrected, healthy and pure,” said Donald Kohn, MD, medical director of the UCLA Human Gene and Cell Therapy Facility. “Previous research studies were limited to producing ½ to 2 million genetically corrected cells, so this represents a significant increase in scale.”

The disease is caused by a single-letter change, or mutation, in human DNA. This trial will use CRISPR genome editing to correct this mutation directly. The project team from UCSF, the IGI, and UCLA have developed CRISPR_SCD001, a patient-specific blood stem cell therapy that has been modified by a CRISPR-Cas9 nuclease to stimulate repair of the sickle mutation.

In the current trial, the patient’s blood stem cells will be extracted and sent to UCLA’s gene manufacturing lab to be processed using electrical pulses that create temporary pores in their membranes. These pores allow the CRISPR-Cas9 platform to enter the cells and travel to the nucleus, where it corrects the sickle cell mutation before the cells are reintroduced to the patient.

“The goal is to develop a cure that is not just safe and effective, but one that is affordable by to those who need it most,” Doudna said. “There have been a lot of promising developments in recent years with CRISPR-based therapies for sickle cell and other genetic diseases, but it’s essential that we have studies like this that can help us create truly accessible cures.”

As part of its legacy, BCH Oakland was recognized for establishing the first not-for-profit sibling donor cord blood bank, leading to children being cured of sickle cell disease and other blood diseases.

Among its many accomplishments in treating sickle cell disease, BCH Oakland scientists studied and received FDA approval for the drug Voxelotor to improve hemoglobin levels and reduce anemia and hemolysis in sickle cell patients.

Going forward, the UCSF research team is hoping to use expanded resources under development in the UCSF Living Therapeutics Initiative as this new treatment is expanded, including the potential to manufacture the investigational cell therapy product at UCSF.

Lorna Fernandes is the senior public information representative at the UCSF Office of Communications and Public Affairs.

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Big God Ministry Gives Away Toys in Marin City

Pastor Hall also gave a message of encouragement to the crowd, thanking Jesus for the “best year of their lives.” He asked each of the children what they wanted to be when they grow up.

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From top left: Pastor David Hall asking the children what they want to be when they grow up. Worship team Jake Monaghan, Ruby Friedman, and Keri Carpenter. Children lining up to receive their presents. Photos by Godfrey Lee.
From top left: Pastor David Hall asking the children what they want to be when they grow up. Worship team Jake Monaghan, Ruby Friedman, and Keri Carpenter. Children lining up to receive their presents. Photos by Godfrey Lee.

By Godfrey Lee

Big God Ministries, pastored by David Hall, gave toys to the children in Marin City on Monday, Dec. 15, on the lawn near the corner of Drake Avenue and Donahue Street.

Pastor Hall also gave a message of encouragement to the crowd, thanking Jesus for the “best year of their lives.” He asked each of the children what they wanted to be when they grew up.

Around 75 parents and children were there to receive the presents, which consisted mainly of Gideon Bibles, Cat in the Hat pillows, Barbie dolls, Tonka trucks, and Lego building sets.

A half dozen volunteers from the Big God Ministry, including Donnie Roary, helped to set up the tables for the toy giveaway. The worship music was sung by Ruby Friedman, Keri Carpenter, and Jake Monaghan, who also played the accordion.

Big God Ministries meets on Sundays at 10 a.m. at the Mill Valley Community Center, 180 Camino Alto, Mill Valley, CA Their phone number is (415) 797-2567.

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First 5 Alameda County Distributes Over $8 Million in First Wave of Critical Relief Funds for Historically Underpaid Caregivers

“Family, Friend, and Neighbor caregivers are lifelines for so many children and families in Alameda County,” said Kristin Spanos, CEO, First 5 Alameda County. “Yet, they often go unrecognized and undercompensated for their labor and ability to give individualized, culturally connected care. At First 5, we support the conditions that allow families to thrive, and getting this money into the hands of these caregivers and families at a time of heightened financial stress for parents is part of that commitment.”

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Costco. Courtesy image.
Costco. Courtesy image.

Family, Friend, and Neighbor Caregivers Can Now Opt Into $4,000 Grants to Help Bolster Economic Stability and Strengthen Early Learning Experiences

By Post Staff

Today, First 5 Alameda County announced the distribution of $4,000 relief grants to more than 2,000 Family, Friend, and Neighbor (FFN) caregivers, totaling over $8 million in the first round of funding. Over the full course of the funding initiative, First 5 Alameda County anticipates supporting over 3,000 FFN caregivers, who collectively care for an estimated 5,200 children across Alameda County. These grants are only a portion of the estimated $190 million being invested into expanding our early childcare system through direct caregiver relief to upcoming facilities, shelter, and long-term sustainability investments for providers fromMeasure C in its first year. This investment builds on the early rollout of Measure C and reflects a comprehensive, system-wide strategy to strengthen Alameda County’s early childhood ecosystem so families can rely on sustainable, accessible care,

These important caregivers provide child care in Alameda County to their relatives, friends, and neighbors. While public benefits continue to decrease for families, and inflation and the cost of living continue to rise, these grants provide direct economic support for FFN caregivers, whose wages have historically been very low or nonexistent, and very few of whom receive benefits. As families continue to face growing financial pressures, especially during the winter and holiday season, these grants will help these caregivers with living expenses such as rent, utilities, supplies, and food.

“Family, Friend, and Neighbor caregivers are lifelines for so many children and families in Alameda County,” said Kristin Spanos, CEO, First 5 Alameda County. “Yet, they often go unrecognized and undercompensated for their labor and ability to give individualized, culturally connected care. At First 5, we support the conditions that allow families to thrive, and getting this money into the hands of these caregivers and families at a time of heightened financial stress for parents is part of that commitment.”

The funding for these relief grants comes from Measure C, a local voter-approved sales tax in Alameda County that invests in young children, their families, communities, providers, and caregivers. Within the first year of First 5’s 5-Year Plan for Measure C, in addition to the relief grants to informal FFN caregivers, other significant investments will benefit licensed child care providers. These investments include over $40 million in Early Care and Education (ECE) Emergency Grants, which have already flowed to nearly 800 center-based and family child care providers. As part of First 5’s 5-Year Plan, preparations are also underway to distribute facilities grants early next year for child care providers who need to make urgent repairs or improvements, and to launch the Emergency Revolving Fund in Spring 2026 to support licensed child care providers in Alameda County who are at risk of closure.

The FFN Relief Grants recognize and support the essential work that an estimated 3,000 FFN caregivers provide to 5,200 children in Alameda County. There is still an opportunity to receive funds for FFN caregivers who have not yet received them.

In partnership with First 5 Alameda County, Child Care Payment Agencies play a critical role in identifying eligible caregivers and leading coordinated outreach efforts to ensure FFN caregivers are informed of and able to access these relief funds.FFN caregivers are eligible for the grant if they receive a child care payment from an Alameda County Child Care Payment Agency, 4Cs of Alameda County, BANANAS, Hively, and Davis Street, and are currently caring for a child 12 years old or younger in Alameda County. Additionally, FFN caregivers who provided care for a child 12 years or younger at any time since April 1, 2025, but are no longer doing so, are also eligible for the funds. Eligible caregivers are being contacted by their Child Care Payment Agency on a rolling basis, beginning with those who provided care between April and July 2025.

“This money is coming to me at a critical time of heightened economic strain,” said Jill Morton, a caregiver in Oakland, California. “Since I am a non-licensed childcare provider, I didn’t think I was eligible for this financial support. I was relieved that this money can help pay my rent, purchase learning materials for the children as well as enhance childcare, buy groceries and take care of grandchildren.”

Eligible FFN caregivers who provided care at any time between April 1, 2025 and July 31, 2025, who haven’t yet opted into the process, are encouraged to check their mail and email for an eligibility letter. Those who have cared for a child after this period should expect to receive communications from their child care payment agency in the coming months. FFN caregivers with questions may also contact the agency they work with to receive child care payments, or the First 5 Alameda help desk, Monday through Friday, from 9 a.m. to 5:00 p.m. PST, at 510-227-6964. The help desk will be closed 12/25/25 – 1/1/26. Additional grant payments will be made on a rolling basis as opt-ins are received by the four child care payment agencies in Alameda County.

Beginning in the second year of Measure C implementation, FFN caregivers who care for a child from birth to age five and receive an Alameda County subsidized voucher will get an additional $500 per month. This amounts to an annual increase of about $6,000 per child receiving a subsidy. Together with more Measure C funding expected to flow back into the community as part of First 5’s 5-Year Plan, investments will continue to become available in the coming year for addressing the needs of childcare providers in Alameda County.

About First 5 Alameda County

First 5 Alameda County builds the local childhood systems and supports needed to ensure our county’s youngest children are safe, healthy, and ready to succeed in school and life.

Our Mission

In partnership with the community, we support a county-wide continuous prevention and early intervention system that promotes optimal health and development, narrows disparities, and improves the lives of children from birth to age five and their families.

Our Vision

Every child in Alameda County will have optimal health, development, and well-being to reach their greatest potential. 

Learn more at www.first5alameda.org.

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Oakland Post: Week of December 24 – 30, 2025

The printed Weekly Edition of the Oakland Post: Week of – December 24 – 30, 2025

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